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Article

(2001) Activating mutations in theWiskott Aldrich syndrome protein may define a sub-group of severe congenital neutropenia (SCN) with specific and unusual laboratory features. Blood , 98 (11) 1842 - ?.

(2001) Genetic incorporation of the SIGYPLP peptide into adenoassociated virus-2 capsids directs efficient and selective gene transfer to the human vascular endothelial cells. Molecular Therapy , 4 174 - 181.

Abel, U; Deichmann, A; Bartholomae, C; Schwarzwaelder, K; Glimm, H; Howe, S; ... Schmidt, M; + view all (2007) Real-Time Definition of Non-Randomness in the Distribution of Genomic Events. PLOS ONE , 2 (6) , Article e570. 10.1371/journal.pone.0000570. Green and gold open access
file

Abeyewickreme, A; Thrasher, AJ; Kinnon, C; (2011) Bone morphogenetic protein-4 (BMP4) up regulates key haematopoietic genes in differentiating embryonic stem cells treated with BMP4 short hairpin RNA. Br J Haematol , 155 (5) 638 - 641. 10.1111/j.1365-2141.2011.08759.x.

Abo, A; Boyhan, A; West, I; Thrasher, AJ; Segal, AW; (1992) Reconstitution of neutrophil NADPH oxidase activity in the cell-free system by four components: p67-phox, p47-phox, p21rac1, and cytochrome b-245. J Biol Chem , 267 (24) 16767 - 16770. Gold open access

Acosta-Saltos, C; Gonitel, R; Chavda, S; Acosta-Saltos, A; Rahim, A; Thrasher, A; ... Raivich, G; + view all (2011) Viral expression of GM-CSF: neuroinflammatory response and effects on regeneration. HUM GENE THER , 22 (10) A95 - A95.

Adamovich, DA; Nakamura, F; Worth, A; Burns, S; Thrasher, AJ; Hartwig, JH; Snapper, SB; (2009) Activating mutations of N-WASP alter Shigella pathogenesis. BIOCHEM BIOPH RES CO , 384 (3) 284 - 289. 10.1016/j.bbrc.2009.04.050.

Ahmed, F; Ings, SJ; Pizzey, A; Fahey, A; Blundell, M; Hardy, SJ; ... Yong, KL; + view all (2001) Cytokine exposure impairs short-term homing of CD34(+) cells in SCID/NOD mice: Influence of cell cycle status. BLOOD , 98 (11) 644A - 644A.

Ahmed, F; Ings, SJ; Pizzey, AR; Blundell, MP; Thrasher, AJ; T Ye, H; ... Yong, KL; + view all (2004) Impaired bone marrow homing of cytokine-activated CD34(+) cells in the NOD/SCID model. BLOOD , 103 (6) 2079 - 2087. 10.1182/blood-2003-06-1770.

Albert, MH; Bittner, TC; Nonoyama, S; Notarangelo, LD; Burns, S; Imai, K; ... Ochs, HD; + view all (2010) X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options. BLOOD , 115 (16) 3231 - 3238. 10.1182/blood-2009-09-239087.

Alexander, BL; Ali, RR; Alton, EW; Bainbridge, JW; Braun, S; Cheng, SH; ... Ylä-Herttuala, S; + view all (2007) Progress and prospects: gene therapy clinical trials (part 1). Gene Ther , 14 (20) 1439 - 1447. 10.1038/sj.gt.3303001.

Alexander, BL; Ali, RR; Alton, EWFW; Bainbridge, JW; Braun, S; Cheng, SH; ... Herttuala, SY; + view all (2007) Progress and prospects: gene therapy clinical trials (part 1) (vol 14, pg 1439, 2007). GENE THER , 14 (24) 1754 - 1754. 10.1038/sj.gt.3303075.

Ali, RR; Reichel, MB; Byrnes, AP; Stephens, CJ; Thrasher, AJ; Baker, D; ... Bhattacharya, SS; + view all (1998) Co-injection of adenovirus expressing CTLA4-Ig prolongs adenovirally mediated lacZ reporter gene expression in the mouse retina. GENE THER , 5 (11) 1561 - 1565.

Ali, RR; Reichel, MB; De Alwis, M; Kanuga, N; Kinnon, C; Levinsky, RJ; ... Thrasher, AJ; + view all (1998) Adeno-associated virus gene transfer to mouse retina. HUM GENE THER , 9 (1) 81 - 86.

Ali, RR; Reichel, MB; de Alwis, M; Kanuga, N; Kinnon, C; Levinsky, RJ; ... Thrasher, AJ; + view all (1998) Adeno-associated virus gene transfer to mouse retina. Human Gene Therapy. Human Gene Therapy , 9 81 - 86.

Ali, RR; Reichel, MB; Thrasher, AJ; Kanuga, N; Hunt, DM; Bhattacharya, SS; (1997) Gene delivery to the RDS mouse. INVEST OPHTH VIS SCI , 38 (4) 1194 - 1194.

Ali, RR; Reichel, MB; Thrasher, AJ; Levinsky, RJ; Kinnon, C; Kanuga, N; ... Bhattacharya, SS; + view all (1996) Gene transfer into the mouse retina mediated by an adeno-associated viral vector. HUM MOL GENET , 5 (5) 591 - 594.

Ali, RR; Reichel, MB; Thrasher, AP; Kanuga, N; Hunt, DM; Bhattacharya, SS; (1996) In vivo gene delivery to the mouse retina. INVEST OPHTH VIS SCI , 37 (3) 41 - 41.

Ali, RR; Sarra, GM; Stephens, C; de Alwis, M; Bainbridge, JWB; Munro, PM; ... Thrasher, AJ; + view all (2000) Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. NAT GENET , 25 (3) 306 - 310.

Ali, RR; Sarra, GM; Stephens, CM; de Alwis, M; Bainbridge, JWB; Thrasher, AJ; (2001) Gene therapy for inherited retinal degeneration. J MED GENET , 38 S26 - S26.

Almarza, E; Zhang, F; Santilli, G; Blundell, MP; Howe, SJ; Thornhill, SI; ... Thrasher, AJ; + view all (2011) Correction of SCID-X1 Using an Enhancerless Vav Promoter. HUM GENE THER , 22 (3) 263 - 270. 10.1089/hum.2010.119.

Alonso-Ferrero, ME; Burke, D; Heales, S; Thrasher, A; Gaspar, HB; (2011) Gene therapy for lysosomal storage diseases using an enhanced lentiviral vector delivery system. HUM GENE THER , 22 (10) A44 - A45.

Ancliff, PJ; Blundell, MP; Cory, GO; Calle, Y; Worth, A; Kempski, H; ... Thrasher, AJ; + view all (2006) Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia. BLOOD , 108 (7) 2182 - 2189.

Ancliff, PJ; Blundell, MP; Gale, RE; Liesner, R; Hann, IM; Thrasher, AJ; Linch, DC; (2001) Activating mutations in theWiskott Aldrich syndrome protein may define a sub-group of severe congenital neutropenia (SCN) with specific and unusual laboratory features. BLOOD , 98 (11) 439A - 440A.

Ancliff, PJ; Thrasher, AJ; Blundell, MP; Cory, GO; Calle, Y; Worth, A; ... Linch, DC; + view all (2006) Two novel activating mutations in the Wiskott-Aldrich syndrome protein result in congenital neutropenia. Blood , 108 (7) 2182 - 2189. 10.1182/blood-2006-01-010249.

Antoniou, M; Montiel-Equihua, C; Monkeviciute, A; Thrasher, A; Gaspar, B; (2009) Development of a lentiviral vector for systemic gene therapy of SCID-ADA. HUMAN GENE THERAPY , 20 (11) 1467 - 1468.

Apolonia, L; Waddington, S; Collins, M; Thrasher, A; Philpott, N; (2006) Development of Non-Integrating and Site-Specifically Integrating Lentiviral Vectors. MOLECULAR THERAPY , 13 S177 - S177.

Apolonia, L; Waddington, SN; Fernandes, C; Ward, NJ; Bouma, G; Blundell, MP; ... Philpott, NJ; + view all (2007) Stable gene transfer to muscle using non-integrating lentiviral vectors. MOL THER , 15 (11) 1947 - 1954. 10.1038/sj.mt.6300281.

Bacchelli, C; Buckland, KF; Buckridge, S; Salzer, U; Schneider, P; Thrasher, AJ; Gaspar, HB; (2011) The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice. J ALLERGY CLIN IMMUN , 127 (5) 1253 - U259. 10.1016/j.jaci.2011.02.037.

Bacchelli, C; Buckridge, S; Thrasher, AJ; Gaspar, HB; (2007) Translational mini-review series on immunodeficiency: Molecular defects in common variable immunodeficiency. CLIN EXP IMMUNOL , 149 (3) 401 - 409.

Bacchelli, C; Buckridge, S; Thrasher, AJ; Gaspar, HB; (2007) Translational Mini-Review Series on Immunodeficiency:Molecular defects in common variable immunodeficiency. Clinical and Experimental Immunology , 149 (3) 401 - 409. 10.1111/j.1365-2249.2007.03461.x.

Bainbridge, J; Stephens, C; Zhang, Z; Reichel, MB; Thrasher, AJ; Ali, RR; (1999) Efficient transduction of photoreceptor cells using high titre rAAV produced from replicating amplicons and gH- herpes vectors. Investigative Ophthalmology and Visual Science , 40 44747 - ?.

Bainbridge, JW; Mistry, A; De Alwis, M; Paleolog, E; Baker, A; Thrasher, AJ; Ali, RR; (2002) Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. Gene Ther , 9 (5) 320 - 326. 10.1038/sj.gt.3301680.

Bainbridge, JWB; Mistry, A; Binley, K; De Alwis, M; Thrasher, AJ; Naylor, S; Ali, RR; (2003) Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularization. GENE THER , 10 (12) 1049 - 1054. 10.1038/sj.gt.3301945.

Bainbridge, JWB; Mistry, A; De Alwis, M; Paleolog, E; Baker, A; Thrasher, AJ; Ali, RR; (2002) Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. GENE THER , 9 (5) 320 - 326. 10.1038/sj/gt/3301680.

Bainbridge, JWB; Mistry, A; Schlichtenbrede, FC; Smith, A; Broderick, C; De Alwis, M; ... Ali, RR; + view all (2003) Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina. GENE THER , 10 (16) 1336 - 1344. 10.1038/sj.gt.3301990.

Bainbridge, JWB; Mistry, AR; Thrasher, AJ; Ali, RR; (2003) Gene therapy for ocular angiogenesis. CLINICAL SCIENCE , 104 (6) 561 - 575.

Bainbridge, JWB; Smith, AJ; Barker, SS; Robbie, S; Henderson, R; Balaggan, K; ... Ali, RR; + view all (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. NEW ENGL J MED , 358 (21) 2231 - 2239. 10.1056/NEJMoa0802268.

Bainbridge, JWB; Stephens, C; Parsley, K; Demaison, C; Halfyard, A; Thrasher, AJ; Ali, RR; (2001) In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. GENE THER , 8 (21) 1665 - 1668.

Balaggan, KS; Mistry, A; Georgiadis, T; Broderick, C; Bainbridge, J; Thrasher, AJ; Ali, RR; (2004) Inhibition of experimental choroidal neovascularisation by lentivirus mediated delivery of pigment epithelium derived factor. Investigative Ophthalmology and Visual Science , 45 3705 - ?.

Barker, SE; Grosse, SM; Siapati, EK; Kritz, A; Kinnon, C; Thrasher, AJ; Hart, SL; (2007) Immunotherapy for neuroblastoma using syngeneic fibroblasts transfected with IL-2 and IL-12. Br J Cancer , 97 (2) 210 - 217.

Barker, SE; Grosse, SM; Siapati, EK; Kritz, A; Kinnon, C; Thrasher, AJ; Hart, SL; (2007) Immunotherapy for neuroblastoma using syngeneic fibroblasts transfected with IL-2 and IL-12. BRIT J CANCER , 97 (2) 210 - 217. 10.1038/sj.bjc.6603857. Gold open access

Bartholomae, CC; Arens, A; Balaggan, KS; Yanez-Munoz, RJ; Montini, E; Howe, SJ; ... Schmidt, M; + view all (2011) Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues. MOL THER , 19 (4) 703 - 710. 10.1038/mt.2011.19.

Bartholomae, CC; Yanez-Munoz, RJ; Balaggan, KS; Howe, SJ; MacNeil, A; Ali, RR; ... Schmidt, M; + view all (2008) Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. Blood Cells, Molecules, and Diseases , 40 (2) 6 - ?.

Baum, C; Schambach, A; Modlich, U; Thrasher, A; (2007) Gentherapie der SCID-X1. Bundesgesundheitsblatt - Gesundheitsforschung - Gesundheitsschutz , 50 (12) 1507 - 1517. 10.1007/s00103-007-0385-5.

Bigger, BW; Siapati, EK; Mistry, A; Waddington, SN; Nivsarkar, MS; Jacobs, L; ... Themis, M; + view all (2006) Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther , 13 (2) 117 - 126. 10.1038/sj.gt.3302638.

Bigger, BW; Siapati, KE; Mistry, A; Waddington, SN; Nivsarkar, MS; Holder, MV; ... Themis, M; + view all (2004) Induction of immune tolerance and partial phenotypic correction of haermophiliac mice by lentiviral gene transfer of human factor IX to haematopoietic stem cells. The Journal of Gene Medicine , 6 (9) 38 - ?.

Binks, M; Jones, GE; Brickell, PM; Kinnon, C; Katz, DR; Thrasher, AJ; (1998) Intrinsic dendritic cell abnormalities in Wiskott-Aldrich syndrome. EUR J IMMUNOL , 28 (10) 3259 - 3267.

Binks, M; Jones, GE; Brickell, PM; Kinnon, C; Katz, DR; Thrasher, AJ; (1998) Intrinsic dendritic cell defects in Wiskott-Aldrich syndrome. MOL IMMUNOL , 35 (11-12) 734 - 734.

Binley, K; Askham, Z; Iqball, S; Spearman, H; Martin, L; de Alwis, M; ... Naylor, S; + view all (2002) Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy. BLOOD , 100 (7) 2406 - 2413. 10.1182/blood-2002-02-0605.

Binny, C; McIntosh, J; Della Peruta, M; Kymalainen, H; Tuddenham, EGD; Buckley, SMK; ... Nathwani, AC; + view all (2012) AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. BLOOD , 119 (4) 957 - 966. 10.1182/blood-2011-09-377630.

Binny, C; McIntosh, J; Peruta, M; Kymalainen, H; Tuddenham, E; Waddington, S; ... Nathwani, A; + view all (2011) AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. HUMAN GENE THERAPY , 22 (10) A45 - A45.

Binny, C; McIntosh, J; Peruta, M; Kymalainen, H; Tuddenham, E; Waddington, S; ... Nathwani, A; + view all (2011) AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. HUMAN GENE THERAPY , 22 (10) A45 - A45.

Blundell, MP; Bouma, G; Calle, Y; Jones, GE; Kinnon, C; Thrasher, AJ; (2008) Improvement of migratory defects in a murine model of Wiskott-Aldrich syndrome gene therapy. MOL THER , 16 (5) 836 - 844. 10.1038/mt.2008.43.

Blundell, MP; Bouma, G; Calle, Y; Jones, GE; Kinnon, C; Thrasher, AJ; (2008) Restoration of migratory defects in a murine model of WiskottAldrich syndrome gene therapy. HUMAN GENE THERAPY , 19 (4) 400 - 400.

Blundell, MP; Bouma, G; Metelo, J; Worth, A; Calle, Y; Cowell, LA; ... Thrasher, AJ; + view all (2009) Phosphorylation of WASp is a key regulator of activity and. Proc Natl Acad Sci U S A , 106 (37) 15738 - 15743.

Blundell, MP; Bouma, G; Metelo, J; Worth, A; Calle, Y; Cowell, LA; ... Thrasher, AJ; + view all (2009) Phosphorylation of WASp is a key regulator of activity and stability in vivo. P NATL ACAD SCI USA , 106 (37) 15738 - 15743. 10.1073/pnas.0904346106. Gold open access

Blundell, MP; Demaison, C; Brouns, G; Goldman, JP; Gaspar, HB; Kinnon, C; ... Sirchia, G; + view all (1999) Quality of repopulation in nonobese diabetic severe combined immunodeficient mice engrafted with expanded cord blood CD34 cells [1]. Blood , 94 (9) 3269 - 3270.

Blundell, MP; Worth, A; Bouma, G; Thrasher, AJ; (2010) The Wiskott-Aldrich syndrome: The actin cytoskeleton and immune cell function. DIS MARKERS , 29 (3-4) 157 - 175. 10.3233/DMA-2010-0735.

Booth, C; Gaspar, HB; Thrasher, AJ; (2011) Gene therapy for primary immunodeficiency. Curr Opin Pediatr , 23 (6) 659 - 666. 10.1097/MOP.0b013e32834cd67a.

Booth, C; Ribeil, JA; Audat, F; Dal Cortivo, L; Veys, PA; Thrasher, AJ; ... Gaspar, HB; + view all (2006) CD34+stem cell top-ups without conditioning after initial HSCT for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies. Bone Marrow Transplantation , 37 S135 - S135.

Booth, C; Ribeil, JA; Audat, F; Dal-Cortivo, L; Veys, PA; Thrasher, AJ; ... Gaspar, HB; + view all (2006) CD34(+) stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies. BRIT J HAEMATOL , 135 (4) 533 - 537. 10.1111/j.1365-2141.2006.06333.x.

Booth, MJ; Coffin, RS; Mistry, A; Thrasher, A; Li, X; (2004) Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids. Gene Therapy , 11 (10) 829 - 837. 10.1038/sj.gt.3302226.

Booth, MJ; Mistry, A; Li, X; Thrasher, A; Coffin, RS; (2004) Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids. GENE THER , 11 (10) 829 - 837. 10.1038/sj.gt.3302226.

Boucherie, C; Mukherjee, S; Henckaerts, E; Thrasher, AJ; Sowden, JC; Ali, RR; (2013) Brief report: self-organizing neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptors. Stem Cells , 31 (2) 408 - 414. 10.1002/stem.1268.

Bouma, G; Ancliff, PJ; Thrasher, AJ; Burns, SO; (2010) Recent advances in the understanding of genetic defects of neutrophil number and function. BRIT J HAEMATOL , 151 (4) 312 - 326. 10.1111/j.1365-2141.2010.08361.x.

Bouma, G; Burns, S; Thrasher, AJ; (2007) Impaired T-cell priming in vivo resulting from dysfunction of WASp-deficient dendritic cells. BLOOD , 110 (13) 4278 - 4284. 10.1182/blood-2007-06-096875.

Bouma, G; Burns, SO; Thrasher, AJ; (2009) Wiskott-Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation. IMMUNOBIOLOGY , 214 (9-10) 778 - 790. 10.1016/j.imbio.2009.06.009. Gold open access

Bouma, G; Burns, SO; Thrasher, AJ; (2009) Wiskott-Aldrich Syndrome: Immunodeficiency resulting from. Immunobiology , 214 (9-10) 778 - 790.

Bouma, G; Carter, NA; Recher, M; Malinova, D; Adriani, M; Notarangelo, LD; ... Thrasher, AJ; + view all (2014) Exacerbated experimental arthritis in Wiskott-Aldrich syndrome protein deficiency: Modulatory role of regulatory B cells. Eur J Immunol 10.1002/eji.201344245.

Bouma, G; Doffinger, R; Patel, SY; Peskett, E; Sinclair, JC; Barcenas-Morales, G; ... Burns, SO; + view all (2009) Impaired neutrophil migration and phagocytosis in IRAK-4 deficiency. BRIT J HAEMATOL , 147 (1) 153 - 156. 10.1111/j.1365-2141.2009.07838.x.

Bouma, G; Mendoza-Naranjo, A; Blundell, MP; de Falco, E; Parsley, KL; Burns, SO; Thrasher, AJ; (2011) Cytoskeletal remodeling mediated by WASp in dendritic cells is necessary for normal immune synapse formation and T-cell priming. BLOOD , 118 (9) 2492 - 2501. 10.1182/blood-2011-03-340265.

Bouma, G; Peskett, E; Sinclair, JC; Graham Davies, E; Thrasher, AJ; Burns, SO; ... Barcenas-Morales, G; + view all (2009) Impaired neutrophil migration and phagocytosis in IRAK-4 deficiency: Correspondence. British Journal of Haematology , 147 (1) 153 - 156. 10.1111/j.1365-2141.2009.07838.x.

Bovellan, M; Romeo, Y; Biro, M; Boden, A; Chugh, P; Yonis, A; ... Charras, G; + view all (2014) Cellular control of cortical actin nucleation. Curr Biol , 24 (14) 1628 - 1635. 10.1016/j.cub.2014.05.069.

Brickell, PM; Katz, DR; Thrasher, AJ; (1998) Wiskott-Aldrich syndrome: Current research concepts. BRIT J HAEMATOL , 101 (4) 603 - 608.

Broderick, CA; Smith, AJ; Balaggan, KS; Georgarias, A; Buch, PK; Trittibach, PC; ... Ali, RR; + view all (2005) Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis. MOL THER , 12 (2) 369 - 373. 10.1016/j.ymthe.2005.03.018.

Broderick, CA; Smith, AJ; Balaggan, KS; Georgiadis, A; Buch, PK; Trittibach, PC; ... Dick, AD; + view all (2006) Erratum: "Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis" (Molecular Therapy (2005) vol. 12 (369-373) 10.1016/j.ymthe.2005.03.018). Molecular Therapy , 13 (4) 829 - 829. 10.1016/j.ymthe.2005.09.013.

Broderick, CA; Smith, AJ; Balaggan, KS; Georgiadis, A; Buch, PK; Trittibach, PC; ... Ali, RR; + view all (2006) Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis (vol 12, pg 369, 2005). MOL THER , 13 (4) 829 - 829. 10.1016/j.ymthe.2005.03.018.

Brouns, G; Demaison, C; Blundell, M; Goldman, J; Levinsky, RJ; Grez, M; ... Thrasher, AJ; + view all (1999) Evidence for human myeloid-restricted haematopoietic stem cells. British Journal of Haematology , 105 284 - ?.

Brown, JR; Goldblatt, D; Buddle, J; Morton, L; Thrasher, AJ; (2003) Diminished production of anti-inflammatory mediators during neutrophil apoptosis and macrophage phagocytosis in chronic granulomatous disease (CGD). J LEUKOCYTE BIOL , 73 (5) 591 - 599.

Buckley, SM; Howe, SJ; Wong, SP; Buning, H; McIntosh, J; Baker, A; ... Waddington, S; + view all (2008) Luciferin detection after intra-nasal vector delivery is improved by intra-nasal rather than intra-peritoneal luciferin administration. Hum Gene Ther 10.1089/hgt.2008.023.

Buckley, SMK; Howe, SJ; Rahim, AA; Buning, H; McIntosh, J; Wong, SP; ... Waddington, SN; + view all (2008) Luciferin Detection After Intranasal Vector Delivery Is Improved by Intranasal Rather Than Intraperitoneal Luciferin Administration. HUM GENE THER , 19 (10) 1050 - 1056. 10.1089/hum.2008.023.

Buckley, SMK; Howe, SJ; Sheard, V; Ward, NJ; Coutelle, C; Thrasher, AJ; ... Mckay, TR; + view all (2008) Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. GENE THER , 15 (16) 1167 - 1175. 10.1038/gt.2008.74.

Burns, S; Cory, GO; Vainchenker, W; Thrasher, AJ; (2004) Mechanisms of WASp-mediated hematologic and immunologic disease. BLOOD , 104 (12) 3454 - 3462. 10.1182/blood-2004-04-1678.

Burns, S; Hardy, SJ; Buddle, J; Yong, KL; Jones, GE; Thrasher, AJ; (2004) Maturation of DC is associated with changes in motile characteristics and adherence. CELL MOTIL CYTOSKEL , 57 (2) 118 - 132. 10.1002/cm.10163.

Burns, S; Thrasher, AJ; (2004) Dendritic cells: The bare bones of immunity. CURR BIOL , 14 (22) R965 - R967. 10.1016/j.cub.2004.10.044.

Burns, S; Thrasher, AJ; Blundell, MP; Machesky, L; Jones, GE; (2001) Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation. BLOOD , 98 (4) 1142 - 1149.

Burns, SO; Killock, DJ; Moulding, DA; Metelo, J; Nunes, J; Taylor, RR; ... Ivetic, A; + view all (2010) A congenital activating mutant of WASp causes altered plasma membrane topography and adhesion under flow in lymphocytes. BLOOD , 115 (26) 5355 - 5365. 10.1182/blood-2009-08-236174.

Burns, SO; McLatchie, A; Gutierrez, BM; Hassan, A; Jones, AM; Malone, M; ... Nejentsev, S; + view all (2014) Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ. Journal of Allergy and Clinical Immunology , 134 (1) 10.1016/j.jaci.2013.12.1093.

Burns, SO; Plagnol, V; Gutierrez, BM; Al Zahrani, D; Curtis, J; Gaspar, M; ... Nejentsev, S; + view all (2014) Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ(⋆). J Allergy Clin Immunol 10.1016/j.jaci.2013.12.1093.

Burns, SO; Thrasher, AJ; Zenner, HL; Curtis, J; Nejentsev, S; Plagnol, V; ... Doffinger, R; + view all (2012) LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. Journal of Allergy and Clinical Immunology , 130 (6) 1428 - 1432. 10.1016/j.jaci.2012.07.035.

Burns, SO; Zenner, HL; Plagnol, V; Curtis, J; Mok, K; Eisenhut, M; ... Nejentsev, S; + view all (2012) LRBA gene deletion in a patient presenting with autoimmunity without hypogammaglobulinemia. J Allergy Clin Immunol , 130 (6) 1428 - 1432. 10.1016/j.jaci.2012.07.035.

Calle, Y; Anton, IM; Thrasher, AJ; Jones, GE; (2008) WASP and WIP regulate podosomes in migrating leukocytes. JOURNAL OF MICROSCOPY-OXFORD , 231 (3) 494 - 505.

Calle, Y; Carragher, NO; Thrasher, AJ; Jones, GE; (2006) Inhibition of calpain stabilises podosomes and impairs dendritic cell motility. J CELL SCI , 119 (11) 2375 - 2385. 10.1242/jcs.02939.

Calle, Y; Chou, HC; Thrasher, AJ; Jones, GE; (2004) Wiskott-Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells. J PATHOL , 204 (4) 460 - 469. 10.1002/path.1651.

Calle, Y; Jones, GE; Jagger, C; Fuller, K; Blundell, MP; Chow, J; ... Thrasher, AJ; + view all (2004) WASp deficiency in mice results in failure to form osteoclast sealing zones and defects in bone resorption. BLOOD , 103 (9) 3552 - 3561.

Carbonaro, DA; Jin, X; Geiger, S; Cooper, A; Kaufman, ML; Hollis, RP; ... Cornetta, K; + view all (2014) Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Molecular Therapy , 22 (3) 607 - 622. 10.1038/mt.2013.265.

Carbonaro, DA; Zhang, L; Jin, X; Montiel-Equihua, C; Geiger, S; Carmo, M; ... Gaspar, HB; + view all (2013) Pre-clinical demonstration of lentiviral vector mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther 10.1038/mt.2013.265.

Carmo, M; Montiel-Equihua, CA; Schambach, A; Baum, C; Thrasher, AJ; Gaspar, HB; (2011) Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. HUM GENE THER , 22 (10) A46 - A47.

Cattoglio, C; Sartori, D; Facchini, G; Recchia, A; Mavilio, F; Antonelli, A; ... Thrasher, AJ; + view all (2007) Hot spots of retroviral integration in human CD34 hematopoietic cells. Blood , 110 (6) 1770 - 1778. 10.1182/blood-2007-01-068759.

Cavazzana-Calvo, M; Hacein-Bey-Abina, S; Thrasher, AJ; Leboulch, P; Fischer, A; (2006) Correction of genetic blood defects by gene transfer. Principles of Molecular Medicine 854 - 861. 10.1007/978-1-59259-963-9_88.

Cavazzana-Calvo, M; Thrasher, A; Mavillo, F; (2004) The future of gene therapy. Nature , 427 (6977) 779 - 781. 10.1038/427779a.

Chan, E; Schaller, T; Eddaoudi, A; Zhan, H; Tan, CP; Jacobsen, M; ... Qasim, W; + view all (2012) Lentiviral Gene Therapy Against Human Immunodeficiency Virus Type 1, Using a Novel Human TRIM21-Cyclophilin A Restriction Factor. HUMAN GENE THERAPY , 23 (11) 1176 - 1185. 10.1089/hum.2012.083.

Chan, E; Schaller, T; Thrasher, A; Towers, G; Qasim, W; (2011) Lentivirus mediated protection against HIV-1 using a human TRIM21-Cyclophilin A fusion protein. HUM GENE THER , 22 (10) A68 - A68.

Chandra, A; Webster, D; Gilmour, KC; Kumararatne, DS; Thrasher, A; (2006) Suspected atypical common gamma chain severe combined immunodeficiency. Journal of Allergy and Clinical Immunology , 117 (2) S288 - S288.

Chandrashekran, A; Isa, I; Dudhia, J; Thrasher, AJ; Dibb, N; Casimir, C; ... Winston, R; + view all (2014) Lentiviral vector transduction of spermatozoa as a tool for the study of early development. FEBS Open Bio , 4 266 - 275. 10.1016/j.fob.2014.02.008. Gold open access

Chandrashekran, A; Sarkar, R; Thrasher, A; Fraser, SE; Dibb, N; Casimir, C; ... Readhead, C; + view all (2014) Efficient generation of transgenic mice by lentivirus-mediated modification of spermatozoa. FASEB JOURNAL , 28 (2) 569 - 576. 10.1096/fj.13-233999.

Charrier, S; Blundell, M; Cedrone, G; Louache, F; Vainchenker, W; Thrasher, AJ; Galy, A; (2013) Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor. HAEMATOLOGICA , 98 (8) 1300 - 1308. Gold open access

Charrier, S; Dupre, L; Blundell, M; Scaramuzza, S; Jeanson, L; Vainchenker, W; ... Galy, A; + view all (2005) Functional correction of lymphocytes and dendritic cells of patients with WAS or XLT, using lentiviral vectors targeting the expression of WAS protein to hematopoietic cells. Blood , 106 (11) 852A - 852A.

Charrier, S; Dupre, L; Scaramuzza, S; Jeanson-Leh, L; Blundell, MP; Danos, O; ... Galy, A; + view all (2007) Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther. , 14 (5) 415 - 428.

Charrier, S; Jeanson-Leh, L; Galy, A; Dupré, L; Scaramuzza, S; Cattaneo, F; ... Eckenberg, R; + view all (2007) Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Therapy , 14 (5) 415 - 428. 10.1038/sj.gt.3302863.

Chetty, M; Thrasher, AJ; Abo, A; Casimir, CM; (1995) Low NADPH oxidase activity in Epstein-Barr-virus-immortalized B-lymphocytes is due to a post-transcriptional block in expression of cytochrome b558. Biochem J , 306 ( Pt 1) 141 - 145.

Chick, HE; Nowrouzi, A; Fronza, R; McDonald, RA; Kane, NM; Alba, R; ... Baker, AH; + view all (2012) Integrase-Deficient Lentiviral Vectors Mediate Efficient Gene Transfer to Human Vascular Smooth Muscle Cells with Minimal Genotoxic Risk. HUMAN GENE THERAPY , 23 (12) 1247 - 1257. 10.1089/hum.2012.042.

Chou, HC; Anton, IM; Holt, MR; Curcio, C; Lanzardo, S; Worth, A; ... Calle, Y; + view all (2006) WIP regulates the stability and localization of WASP to podosomes in migrating dendritic cells. CURR BIOL , 16 (23) 2337 - 2344. 10.1016/j.cub.2006.10.037.

Coffin, RS; Thomas, SK; Thomas, NSB; Lilley, CE; Pizzey, AR; Griffiths, CH; ... Latchman, DS; + view all (1998) Pure populations of transduced primary human cells can be produced using GFP expressing herpes virus vectors and flow cytometry. GENE THER , 5 (5) 718 - 722.

Coffin, RS; Thomas, SK; Thomas, NSB; Lilley, CE; Pizzey, AR; Griffiths, CH; ... Latchman, DS; + view all (1998) Populations of transduced primary human cells can be produced using GFP expressing herpes virus vectors and flow cytometry. Gene Therapy , 5 718 - 722.

Cole, TS; Jones, LKR; McGrogan, P; Pearce, MS; Flood, TJ; Cant, AJ; ... Titman, P; + view all (2012) Emotional and behavioural difficulties in chronic granulomatous disease. ARCHIVES OF DISEASE IN CHILDHOOD , 97 (1) 87 - 87. 10.1136/archdischild-2011-300780.

Cooper, N; Davies, EG; Thrasher, AJ; (2009) Repeated courses of rituximab for autoimmune cytopenias may precipitate profound hypogammaglobulinaemia requiring replacement intravenous immunoglobulin. British Journal of Haematology , 146 (1) 120 - 122. 10.1111/j.1365-2141.2009.07715.x.

Cooray, S; Gilmour, KC; Parsley, KL; Adams, S; Howe, SJ; Al Ghonaium, A; ... Thrasher, AJ; + view all (2011) Persistence of a polyclonal T-cell repertoire in patients with SCID-X1 following gene therapy. HUM GENE THER , 22 (10) A42 - A42.

Cooray, S; Gilmour, KC; Parsley, KL; Zhang, F; Bjorkegren, E; Bayford, J; ... Gaspar, HB; + view all (2011) Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. HUM GENE THER , 22 (10) A40 - A40.

Cooray, S; Howe, SJ; Thrasher, AJ; (2012) Retrovirus and lentivirus vector design and methods of cell conditioning. Methods Enzymol , 507 29 - 57. 10.1016/B978-0-12-386509-0.00003-X.

Davies, EG; Thrasher, A; Lacey, K; Harper, J; (1999) Topical cidofovir for severe molluscum contagiosum. Lancet , 353 (9169) 2042 - ?.

Davies, EG; Thrasher, AJ; (2010) Update on the hyper immunoglobulin M syndromes. Br J Haematol , 149 (2) 167 - 180. 10.1111/j.1365-2141.2010.08077.x.

de Noronha, S; Hardy, S; Sinclair, J; Blundell, MP; Strid, J; Schulz, O; ... Thrasher, AJ; + view all (2005) Impaired dendritic-cell homing in vivo in the absence of Wiskott-Aldrich syndrome protein. BLOOD , 105 (4) 1590 - 1597. 10.1182/blood-2004-06-2332.

de Noronha, S; Hardy, S; Sinclair, J; Blundell, MP; Strid, J; Schulz, O; ... Thrasher, AJ; + view all (2005) Impaired dendritic-cell homing in vivo in the absence of Wiskott-Aldrich syndrome protein. Blood , 105 (4) 1590 - 1597. 10.1182/blood-2004-06-2332.

Deichman, A; Schmidt, M; Abina, SHB; Cavazzana-Calvo, M; Schwarzwaelder, K; Howe, SJ; ... von Kalle, CN; + view all (2008) Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies. Blood Cells, Molecules, and Diseases , 40 (2) 23 - ?.

Deichmann, A; Brugman, MH; Bartholomae, CC; Schwarzwaelder, K; Verstegen, MMA; Howe, SJ; ... Wagemaker, G; + view all (2011) Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy. MOL THER , 19 (11) 2031 - 2039. 10.1038/mt.2011.178.

Deichmann, A; Hacein-Bey-Abina, S; Schmidt, M; Garrigue, A; Brugman, MH; Hu, J; ... Cavazzana-Calvo, M; + view all (2007) Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J CLIN INVEST , 117 (8) 2225 - 2232. 10.1172/JCI31659.

Demaison, C; Brouns, G; Blundell, MP; Goldman, JP; Levinsky, RJ; Grez, M; ... Thrasher, AJ; + view all (2000) A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus-pseudotyped retroviral vector. HUM GENE THER , 11 (1) 91 - 100.

Demaison, C; Brouns, G; Blundell, MP; Goldman, JP; Levinsky, RJ; Kinnon, C; ... Grez, M; + view all (2000) A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus- pseudotyped retroviral vector. Human Gene Therapy , 11 (1) 91 - 100. 10.1089/10430340050016184.

Demaison, C; Parsley, K; Brouns, G; Scherr, M; Battmer, K; Kinnon, C; ... Thrasher, AJ; + view all (2002) High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. HUM GENE THER , 13 (7) 803 - 813.

Di, WL; Archer, F; Semenova, E; Talbot, GE; Harper, JI; Del Rio, M; ... Qasim, W; + view all (2010) Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome derived skin grafts. JOURNAL OF INVESTIGATIVE DERMATOLOGY , 130 S75 - S75.

Di, WL; Larcher, F; Semenova, E; Talbot, GE; Harper, JI; Del Rio, M; ... Qasim, W; + view all (2011) Ex-vivo Gene Therapy Restores LEKTI Activity and Corrects the Architecture of Netherton Syndrome-derived Skin Grafts. MOL THER , 19 (2) 408 - 416. 10.1038/mt.2010.201.

Di, WL; Mellerio, JE; Bernadis, C; Harper, J; Abdul-Wahab, A; Ghani, S; ... Qasim, W; + view all (2013) Phase I study protocol for ex vivo lentiviral gene therapy for the inherited skin disease, netherton syndrome. Hum Gene Ther Clin Dev , 24 (4) 182 - 190. 10.1089/humc.2013.195.

Di, WL; Mellerio, JE; Bernadis, C; Harper, J; Abdul-Wahab, A; Ghani, S; ... Qasim, W; + view all (2013) Phase I study protocol for ex-vivo lentiviral gene therapy for the inherited skin disease, Netherton Syndrome. Hum Gene Ther Clin Dev 10.1089/hum.2013.195.

Di, WL; Semenova, E; Larcher, F; Del Rio, M; Harper, JI; Thrasher, AJ; Qasim, W; (2012) Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression. Hum Gene Ther , 23 (1) 83 - 90. 10.1089/hum.2011.091.

Fabes, J; Anderson, P; Yáñez-Muñoz, RJ; Thrasher, A; Brennan, C; Bolsover, S; (2006) Accumulation of the inhibitory receptor EphA4 may prevent regeneration of corticospinal tract axons following lesion. Eur J Neurosci , 23 (7) 1721 - 1730. 10.1111/j.1460-9568.2006.04704.x.

Feudner, E; de Alwis, M; Thrasher, AJ; Ali, RR; Fauser, S; (2001) Optimization of recombinant adeno-associated virus production using an herpes simplex virus amplicon system. J VIROL METHODS , 96 (2) 97 - 105.

Field, AC; Vink, C; Gabriel, R; Al-Subki, R; Schmidt, M; Goulden, N; ... Qasim, W; + view all (2013) Comparison of Lentiviral and Sleeping Beauty Mediated αβ T Cell Receptor Gene Transfer. PLoS One , 8 (6) , Article e68201. 10.1371/journal.pone.0068201. Green and gold open access
file

Fischer, A; Abina, SH; Thrasher, A; Von Kalle, C; Cavazzana-Calvo, M; (2004) LMO2 and gene therapy for severe combined immunodeficiency. New England Journal of Medicine , 350 (24) 2526 - 2527.

Fischer, A; Hacein-Bey Abina, S; Cavazzana-Calvo, M; Thrasher, A; Von Kalle, C; Stone, BD; ... McCormack, MP; + view all (2004) LMO2 and gene therapy for severe combined immunodeficiency [7] (multiple letters). New England Journal of Medicine , 350 (24) 2526 - 2527. 10.1056/NEJM200406103502422.

Furmanski, AL; O'Shaughnessy, RF; Saldana, JI; Blundell, MP; Thrasher, AJ; Sebire, NJ; ... Crompton, T; + view all (2013) T-cell reconstitution after thymus xenotransplantation induces hair depigmentation and loss. Journal of Investigative Dermatology , 133 (5) 1221 - 1230. 10.1038/jid.2012.492. Green and gold open access
file

Gabriel, R; Eckenberg, R; Paruzynski, A; Bartholomae, CC; Nowrouzi, A; Arens, A; ... Schmidt, M; + view all (2009) Comprehensive genomic access to vector integration in clinical gene therapy. NAT MED , 15 (12) 1431 - U13. 10.1038/nm.2057.

Gabriel, R; Paruzynski, A; Eckenberg, R; Bartholomae, CC; Nowrouzi, A; Wang, W; ... Schmidt, M; + view all (2008) Assessing the genome accessibility of viral integration sites. HUMAN GENE THERAPY , 19 (10) 1085 - 1085.

Galy, A; Roncarolo, M-G; Thrasher, AJ; (2008) Development of lentiviral gene therapy for Wiskott Aldrich syndrome. Expert Opinion on Biological Therapy , 8 (2) 181 - 190. 10.1517/14712598.8.2.181.

Galy, A; Thrasher, AJ; (2011) Gene therapy for the Wiskott-Aldrich syndrome. CURR OPIN ALLERGY CL , 11 (6) 545 - 550. 10.1097/ACI.0b013e32834c230c.

Gaspar, B; Thrasher, A; (2007) Successful bone marrow gene therapy for severe combined immunodeficiency. Inmunologia , 26 (4) 218 - 219.

Gaspar, HB; Bjorkegren, E; Parsley, K; Gilmour, KC; King, D; Sinclair, J; ... Thrasher, AJ; + view all (2006) Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. MOL THER , 14 (4) 505 - 513. 10.1016/j.ymthe.2006.06.007.

Gaspar, HB; Cooray, S; Gilmour, KC; Parsley, KL; Adams, S; Howe, SJ; ... Thrasher, AJ; + view all (2011) Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency. SCI TRANSL MED , 3 (97) , Article 97ra79. 10.1126/scitranslmed.3002715.

Gaspar, HB; Cooray, S; Gilmour, KC; Parsley, KL; Howe, SJ; Bayford, J; ... Al Ghonaium, A; + view all (2011) Immunodeficiency: Long-term persistence of a polyclonal t cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Science Translational Medicine , 3 (97) 10.1126/scitranslmed.3002715.

Gaspar, HB; Cooray, S; Gilmour, KC; Parsley, KL; Zhang, F; Adams, S; ... Thrasher, AJ; + view all (2011) Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction. SCI TRANSL MED , 3 (97) , Article 97ra80. 10.1126/scitranslmed.3002716.

Gaspar, HB; Cooray, S; Gilmour, KC; Parsley, KL; Zhang, F; Bjorkegren, E; ... Petropolou, T; + view all (2011) Immunodeficiency: Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Science Translational Medicine , 3 (97) 10.1126/scitranslmed.3002716.

Gaspar, HB; Howe, S; Thrasher, AJ; (2003) Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency. Gene Ther , 10 (24) 1999 - 2004. 10.1038/sj.gt.3302150.

Gaspar, HB; Parsley, KL; Howe, S; King, D; Gilmour, KC; Sinclair, J; ... Thrasher, AJ; + view all (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. LANCET , 364 (9452) 2181 - 2187.

Gaspar, HB; Parsley, KL; Howe, S; King, D; Gilmour, KC; Sinclair, J; ... Thrasher, AJ; + view all (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet , 364 (9452) 2181 - 2187. 10.1016/S0140-6736(04)17590-9.

Gaspar, HB; Sharifi, R; Gilmour, KC; Thrasher, AJ; (2002) X-linked lymphoproliferative disease: Clinical, diagnostic and molecular perspective. BRIT J HAEMATOL , 119 (3) 585 - 595.

Gaspar, HB; Swift, S; Thrasher, AJ; (2013) "Special exemptions": should they be put on trial? Mol Ther , 21 (2) 261 - 262. 10.1038/mt.2013.1.

Gaspar, HB; Thrasher, AJ; (2005) Gene therapy for severe combined immunodeficiencies. Expert Opin Biol Ther , 5 (9) 1175 - 1182. 10.1517/14712598.5.9.1175.

Gaspar, HB; Thrasher, AJ; Harwood, C; Leigh, I; (2004) Severe cutaneous papillomavirus disease after haematopoietic stem-cell transplantation in patients with severe combined immunodeficiency [3]. British Journal of Haematology , 127 (2) 232 - 233. 10.1111/j.1365-2141.2004.05176.x.

Gaspar, HB; White, HN; Veys, PA; Thrasher, AJ; (1999) Molecular analysis of B cell repertoire development in patients with X-linked severe combined immunodeficiency following haematopoietic stem cell transplantation. Bone Marrow Transplantation , 23 580 - ?.

Gennery, AR; Thrasher, AJ; (2004) Gene therapy for severe combined immunodeficiency. Current Anaesthesia and Critical Care , 15 (1) 3 - 9. 10.1016/j.cacc.2004.01.008.

Georgiadis, A; Tschernutter, M; Bainbridge, JWB; Balaggan, KS; Mowat, F; West, EL; ... Ali, RR; + view all (2010) The Tight Junction Associated Signalling Proteins ZO-1 and ZONAB Regulate Retinal Pigment Epithelium Homeostasis in Mice. PLOS ONE , 5 (12) , Article e15730. 10.1371/journal.pone.0015730. Green and gold open access
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Giannakopoulos, A; Stavrou, EF; Zarkadis, I; Athanassiadou, A; Zoumbos, N; Thrasher, AJ; (2009) The Functional Role of S/MARs in Episomal Vectors as Defined by the Stress-Induced Destabilization Profile of the Vector Sequences. Journal of Molecular Biology , 387 (5) 1239 - 1249. 10.1016/j.jmb.2009.02.043.

Gilmour, KC; Cranston, T; Jones, A; Goldblatt, D; Thrasher, A; Kinnon, C; ... Gaspar, HB; + view all (2000) Diagnosis of X-linked lymphoproliferative disease by analysis of SLAM-associated protein expression. EUR J IMMUNOL , 30 (6) 1691 - 1697.

Ginn, S; Liao, S; Dane, A; Smyth, C; Hyman, J; Finnie, J; ... Alexander, I; + view all (2009) Lymphomagenesis in SCID-X1 mice independent of insertional mutagenesis and gamma c over-expression. Human Gene Therapy , 20 (11) 1462 - 1462.

Ginn, SL; Liao, SH; Dane, AP; Zheng, M; Alexander, IE; Hu, M; ... Thrasher, AJ; + view all (2010) Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and γc overexpression. Molecular Therapy , 18 (5) 965 - 976. 10.1038/mt.2010.50.

Gjertsson, I; Laurie, KL; Devitt, J; Howe, SJ; Thrasher, AJ; Holmdahl, R; Gustafsson, K; (2009) Tolerance induction using lentiviral gene delivery delays onset and severity of collagen II arthritis. Mol Ther , 17 (4) 632 - 640. 10.1038/mt.2009.299.

Goldblatt, D; Butcher, J; Thrasher, AJ; Russell-Eggit, I; (1999) Ocular findings in chronic granulomatous disease. The Journal of Pediatrics , 134 780 - 783.

Goldblatt, D; Butcher, J; Thrasher, AJ; Russell-Eggitt, I; (1999) Chorioretinal lesions in patients and carriers of chronic granulomatous disease. J Pediatr , 134 (6) 780 - 783.

Goldblatt, D; Thrasher, AJ; (2000) Chronic granulomatous disease. Clin Exp Immunol , 122 (1) 1 - 9.

Goldman, JP; Blundell, MP; Kinnon, C; DiSanto, JP; Thrasher, AJ; (1998) Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor gamma chain. MOL IMMUNOL , 35 (11-12) 779 - 779.

Goldman, JP; Blundell, MP; Lopes, L; Kinnon, C; Di Santo, JP; Thrasher, AJ; (1998) Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor gamma chain. BRIT J HAEMATOL , 103 (2) 335 - 342.

Grez, M; Reichenbach, J; Schwable, J; Seger, R; Dinauer, MC; Thrasher, AJ; (2011) Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma. MOL THER , 19 (1) 28 - 35. 10.1038/mt.2010.232.

Griera, M; Martin-Villar, E; Banon-Rodríguez, I; Blundell, MP; Jones, GE; Anton, IM; ... Calle, Y; + view all (2014) Integrin linked kinase (ILK) regulates podosome maturation and stability in dendritic cells. Int J Biochem Cell Biol 10.1016/j.biocel.2014.01.021.

Griffith, LM; Cowan, MJ; Notarangelo, LD; Kohn, DB; Puck, JM; Pai, SY; ... on behalf of the workshop participants,; + view all (2013) Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol 10.1016/j.jaci.2013.07.052.

Griffith, LM; Cowan, MJ; Puck, JM; Dvorak, CC; Notarangelo, LD; Kohn, DB; ... Zuniga-Pflucker, JC; + view all (2014) Primary Immune Deficiency Treatment Consortium (PIDTC) report. Journal of Allergy and Clinical Immunology , 133 (2) 10.1016/j.jaci.2013.07.052.

Guasti, L; Prasongchean, W; Kleftouris, G; Mukherjee, S; Thrasher, AJ; Bulstrode, NW; Ferretti, P; (2012) High plasticity of pediatric adipose tissue-derived stem cells: too much for selective skeletogenic differentiation? Stem Cells Transl Med , 1 (5) 384 - 395. 10.5966/sctm.2012-0009.

Hacein-Bey-Abina, S; Caccaveli, L; Touzot, F; Dal-Cortivo, L; Heritier, S; Frange, P; ... Cavazzana-Calvo, M; + view all (2011) Efficacy of gene therapy for X-linked severe combined immunodeficiency: update on trial No.1 follow-up and preliminary results of multicentric collaborative trial No. 2 (Paris - Boston - London). HUMAN GENE THERAPY , 22 (10) A40 - A40.

Hacein-Bey-Abina, S; Le Deist, F; De Villartay, J-P; Fischer, A; Cavazzana-Calvo, M; Carlier, F; ... Graham Davies, E; + view all (2002) Erratum: Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy (New England Journal of Medicine (April 18, 2002) 346 (1185-1193)). New England Journal of Medicine , 347 (8) 613 - 613. 10.1056/NEJM200208223470814.

Hacein-Bey-Abina, S; Le Deist, F; Hue, C; De Villartay, J-P; Fischer, A; Cavazzana-Calvo, M; ... Leiva, L; + view all (2002) Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New England Journal of Medicine , 346 (16) 1185 - 1193. 10.1056/NEJMoa012616.

Hart, SL; Arancibia-Carcamo, CV; Wolfert, MA; Mailhos, C; O'Reilly, NJ; Ali, RR; ... Kinnon, C; + view all (1998) Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector. HUM GENE THER , 9 (4) 575 - 585.

Himoudi, N; Nabarro, S; Anderson, J; Buddle, J; Eddaoudi, A; Thrasher, AJ; (2008) Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells. Journal of Immunology , 181 (9) 6654 - 6663.

Himoudi, N; Nabarro, S; Buddle, J; Eddaoudi, A; Thrasher, AJ; Anderson, J; (2008) Bone marrow-derived IFN-producing killer dendritic cells account for the tumoricidal activity of unpulsed dendritic cells. J Immunol , 181 (9) 6654 - 6663.

Himoudi, N; Nabarro, S; Buddle, J; Eddaoudi, A; Thrasher, AJ; Anderson, J; (2008) Bone Marrow-Derived IFN-Producing Killer Dendritic Cells Account for the Tumoricidal Activity of Unpulsed Dendritic Cells. J IMMUNOL , 181 (9) 6654 - 6663.

Himoudi, N; Nabarro, S; Yan, M; Anderson, J; Gilmour, K; Thrasher, AJ; (2007) Development of anti-PAX3 immune responses; a target for cancer immunotherapy. Cancer Immunology, Immunotherapy , 56 (9) 1381 - 1395. 10.1007/s00262-007-0294-3.

Himoudi, N; Nabarro, S; Yan, M; Gilmour, K; Thrasher, AJ; Anderson, J; (2007) Development of anti-PAX3 immune responses; a target for cancer immunotherapy. CANCER IMMUNOL IMMUN , 56 (9) 1381 - 1395. 10.1007/s00262-007-0294-3.

Himoudi, N; Wallace, R; Parsley, KL; Gilmour, K; Barrie, AU; Howe, K; ... Anderson, J; + view all (2012) Lack of T-cell responses following autologous tumour lysate pulsed dendritic cell vaccination, in patients with relapsed osteosarcoma. Clin Transl Oncol , 14 (4) 271 - 279. 10.1007/s12094-012-0795-1.

Howe, S; Bartolovic, K; Milsom, M; Thrasher, A; Kinnon, C; (2009) Optimisation of integration-deficient lentivirus (IDLV) application in hematopoietic stem cells. HUMAN GENE THERAPY , 20 (11) 1463 - 1463.

Howe, S; Thrasher, A; (2003) Gene therapy for inherited immunodeficiencies. Current Hematology Reports , 2 283 - 334.

Howe, S; Thrasher, AJ; (2003) Gene therapy for inherited immunodeficiencies. Curr Hematol Rep , 2 (4) 328 - 334.

Howe, SJ; Mansour, M; Brugman, M; Pike-Overzet, K; Hubank, M; Jina, N; ... Thrasher, AJ; + view all (2008) Molecular analysis of a severe adverse event in the UKSCID-X1 gene therapy clinical trial. Human Gene Therapy , 19 (4) 82 - ?.

Howe, SJ; Mansour, MR; Schwarzwaelder, K; Bartholomae, C; Hubank, M; Kempski, H; ... Thrasher, AJ; + view all (2008) Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J CLIN INVEST , 118 (9) 3143 - 3150. 10.1172/JCI35798. Gold open access

Howe, SJ; Thornhill, SI; Parsley, KL; Quaye, M; Kinnon, C; Gaspar, HB; ... Webb, DK; + view all (2008) Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. Journal of Clinical Investigation , 118 (9) 3143 - 3150. 10.1172/JCI35798.

Hubbard, VG; Breuer, J; Leigh, IM; Thrasher, A; Harwood, CA; (2004) Viral warts following bone marrow transplantation for severe combined immunodeficiency harbour potentially oncogenic human papillomavirus types. British Journal of Dermatology , 151 19 - 19.

Hudde, T; Rayner, SA; De Alwis, M; Thrasher, AJ; Smith, J; Coffin, RS; ... Larkin, DFP; + view all (2000) Adeno-associated and herpes simplex viruses as vectors for gene transfer to the corneal endothelium. CORNEA , 19 (3) 369 - 373.

Huston, MW; van Til, NP; Visser, TP; Arshad, S; Brugman, MH; Cattoglio, C; ... Wagemaker, G; + view all (2011) Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning. MOL THER , 19 (10) 1867 - 1877. 10.1038/mt.2011.127.

Jones, GE; Zicha, D; Dunn, GA; Blundell, M; Thrasher, A; (2002) Restoration of podosomes and chemotaxis in Wiskott-Aldrich syndrome macrophages following induced expression of WASp. Int J Biochem Cell Biol , 34 (7) 806 - 815.

Jones, LBKR; McGrogan, P; Flood, TJ; Gennery, AR; Morton, L; Thrasher, A; ... Cant, AJ; + view all (2008) Special Article: Chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry. CLINICAL AND EXPERIMENTAL IMMUNOLOGY , 152 (2) 211 - 218. 10.1111/j.1365-2249.2008.03644.x.

Jones, RA; Feng, Y; Worth, AJ; Thrasher, AJ; Burns, SO; Martin, P; (2013) Modelling of human Wiskott-Aldrich syndrome protein mutants in zebrafish larvae using in vivo live imaging. JOURNAL OF CELL SCIENCE , 126 (18) 4077 - 4084. 10.1242/jcs.128728.

Jonuschies, J; Antoniou, M; Waddington, S; Boldrin, L; Muntoni, F; Thrasher, A; Morgan, J; (2014) The Human Desmin Promoter Drives Robust Gene Expression for Skeletal Muscle Stem Cell-Mediated Gene Therapy. Curr Gene Ther

Jonuschies, J; Boldrin, L; Thrasher, A; Morgan, JE; Muntoni, F; (2011) Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER , 22 (10) A5 - A5.

Jonuschies, J; Boldrin, L; Thrasher, A; Morgan, JE; Muntoni, F; (2011) Assessment of potential promoters for lentiviral gene therapy in DMD. HUM GENE THER , 22 (10) A62 - A63.

Kallinikou, K; Anjos-Afonso, F; Blundell, MP; Ings, SJ; Watts, MJ; Thrasher, AJ; ... Yong, KL; + view all (2012) Engraftment defect of cytokine-cultured adult human mobilized CD34(+) cells is related to reduced adhesion to bone marrow niche elements. Br J Haematol , 158 (6) 778 - 787. 10.1111/j.1365-2141.2012.09219.x.

Kane, NM; Nowrouzi, A; Mukherjee, S; Blundell, MP; Greig, JA; Lee, WK; ... Baker, AH; + view all (2010) Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors. MOL THER , 18 (12) 2139 - 2145. 10.1038/mt.2010.231.

Kane, NM; Thrasher, AJ; Angelini, GD; Emanueli, C; (2014) MicroRNAs as modulators of stem cells and angiogenesis. Stem Cells 10.1002/stem.1629.

Kao, V; Waddington, S; Thrasher, A; Antoniou, M; (2008) Novel lentiviral vectors for targeting the red blood cell lineage. HUMAN GENE THERAPY , 19 (4) 415 - 415.

Kao, VYC; Waddington, S; Equihua, CM; Thrasher, AJ; Antoniou, MN; (2011) Curative F.IX production from low-dose UCOE-lentiviral vectors. HUM GENE THER , 22 (10) A110 - A110.

Karwacz, K; Mukherjee, S; Apolonia, L; Blundell, MP; Bouma, G; Escors, D; ... Thrasher, AJ; + view all (2009) Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy. J.Virol. , 83 (7) 3094 - 3103.

Karwacz, K; Mukherjee, S; Apolonia, L; Blundell, MP; Bouma, G; Escors, D; ... Thrasher, AJ; + view all (2009) Nonintegrating Lentivector Vaccines Stimulate Prolonged T-Cell and Antibody Responses and Are Effective in Tumor Therapy. J VIROL , 83 (7) 3094 - 3103. 10.1128/JVI.02519-08.

Karwacz, K; Mukherjee, S; Apolonia, L; Blundell, MP; Bouma, G; Escors, D; ... Thrasher, AJ; + view all (2009) Non-integrating Lentivector Vaccines Stimulate Prolonged T Cell and Antibody Responses and are Effective in Tumor Therapy. J.Virol.

Kim, JJ; Thrasher, AJ; Jones, AM; Davies, EG; Cale, CM; (2007) Rituximab for the treatment of autoimmune cytopenias in children with immune deficiency. Br J Haematol , 138 (1) 94 - 96. 10.1111/j.1365-2141.2007.06616.x.

Knight, S; Zhang, F; Mueller-Kuller, U; Bokhoven, M; Gupta, A; Broughton, T; ... Takeuchi, Y; + view all (2012) Safer, silencing-resistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing. J Virol , 86 (17) 9088 - 9095. 10.1128/JVI.00485-12. Gold open access

Lang, PA; Brenner, D; Mak, TW; Ohashi, PS; Shaabani, N; Honke, N; ... Bouma, G; + view all (2013) Reduced type I interferon production by dendritic cells and weakened antiviral immunity in patients with Wiskott-Aldrich syndrome protein deficiency. Journal of Allergy and Clinical Immunology , 131 (3) 815-824.e2. 10.1016/j.jaci.2012.08.050. Green and gold open access
file

Langrish, CL; Buddle, JC; Thrasher, AJ; Goldblatt, D; (2002) Neonatal dendritic cells are intrinsically biased against Th-1 immune responses. CLIN EXP IMMUNOL , 128 (1) 118 - 123.

Laurie, KL; Blundell, MP; Baxendale, HE; Howe, SJ; Sinclair, J; Qasim, W; ... Gustafsson, K; + view all (2007) Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector. GENE THER , 14 (23) 1623 - 1631. 10.1038/sj.gt.3303021.

Leverrier, Y; Lorenzi, R; Blundell, MP; Brickell, P; Kinnon, C; Ridley, AJ; Thrasher, AJ; (2001) Cutting edge: The Wiskott-Aldrich syndrome protein is required for efficient phagocytosis of apoptotic cells. J IMMUNOL , 166 (8) 4831 - 4834.

LEVIN, M; NEWPORT, MJ; DSOUZA, S; KALABALIKIS, P; BROWN, IN; LENICKER, HM; ... BLACKWELL, JM; + view all (1995) FAMILIAL DISSEMINATED ATYPICAL MYCOBACTERIAL INFECTION IN CHILDHOOD - A HUMAN MYCOBACTERIAL SUSCEPTIBILITY GENE. LANCET , 345 (8942) 79 - 83.

Li, JM; Mullen, AM; Yun, S; Wientjes, F; Brouns, GY; Thrasher, AJ; Shah, AM; (2002) Essential role of the NADPH oxidase subunit p47(phox) in endothelial cell superoxide production in response to phorbol ester and tumor necrosis factor-alpha. CIRC RES , 90 (2) 143 - 150.

Locci, M; Draghici, E; Marangoni, F; Bosticardo, M; Catucci, M; Aiuti, A; ... Villa, A; + view all (2009) The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function. J EXP MED , 206 (4) 735 - 742. 10.1084/jem.20081773.

Loew, R; Meyer, Y; Kuehlcke, K; Gama-Norton, L; Wirth, D; Hauser, H; ... Schambach, A; + view all (2010) A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating gamma-retroviral vectors using targeted integration. GENE THERAPY , 17 (2) 272 - 280. 10.1038/gt.2009.134.

Lorenzi, R; Brickell, PM; Katz, DR; Kinnon, C; Thrasher, AJ; (2000) Wiskott-Aldrich syndrome protein is necessary for efficient IgG-mediated phagocytosis. BLOOD , 95 (9) 2943 - 2946.

Lowdell, MW; Birchall, M; Thrasher, AJ; (2012) Use of compassionate-case ATMP in preclinical data for clinical trial applications. Lancet , 379 (9834) 2341 - ?. 10.1016/S0140-6736(12)61018-6.

Luevano, M; Domogala, A; Blundell, M; Jackson, N; Pedroza-Pacheco, I; Derniame, S; ... Saudemont, A; + view all (2014) Frozen cord blood hematopoietic stem cells differentiate into higher numbers of functional natural killer cells in vitro than mobilized hematopoietic stem cells or freshly isolated cord blood hematopoietic stem cells. PLoS One , 9 (1) e87086 - ?. 10.1371/journal.pone.0087086.

MacLaren, RE; Ali, RR; Thrasher, AJ; (2005) Risks of gene therapy should be weighed against lack of alternatives for many diseases. BRIT MED J , 330 (7494) 791 - 791.

Macpherson, L; Monypenny, J; Blundell, MP; Cory, GO; Tomé-García, J; Thrasher, AJ; ... Calle, Y; + view all (2012) Tyrosine phosphorylation of WASP promotes calpain-mediated podosome disassembly. Haematologica , 97 (5) 687 - 691. 10.3324/haematol.2011.048868.

Mandegar, MA; Moralli, D; Khoja, S; Cowley, S; Chan, DYL; Yusuf, M; ... Monaco, ZL; + view all (2011) Functional human artificial chromosomes are generated and stably maintained in human embryonic stem cells. HUM MOL GENET , 20 (15) 2905 - 2913. 10.1093/hmg/ddr144.

Marden, C; Graham, DC; Casimir, C; Thrasher, A; (2003) A functional ISRE is required for myeloid transcription of the p47 gene. Biochimica et Biophysica Acta - Gene Structure and Expression , 1630 (2-3) 117 - 122. 10.1016/j.bbaexp.2003.09.005.

Marshall, CJ; Kinnon, C; Thrasher, AJ; (2000) Polarized expression of bone morphogenetic protein-4 in the human aorta-gonad-mesonephros region. BLOOD , 96 (4) 1591 - 1593.

Marshall, CJ; Moore, RL; Thorogood, P; Brickell, PM; Kinnon, C; Thrasher, AJ; (1999) Detailed characterization of the human aorta-gonad-mesonephros region reveals morphological polarity resembling a hematopoietic stromal layer. DEV DYNAM , 215 (2) 139 - 147.

Marshall, CJ; Sinclair, JC; Thrasher, AJ; Kinnon, C; (2007) Bone morphogenetic protein 4 modulates c-Kit expression and differentiation potential in murine embryonic aorta-gonad-mesonephros haematopoiesis in vitro. BRIT J HAEMATOL , 139 (2) 321 - 330. 10.1111/j.1365-2141.2007.06795.x. Gold open access

Marshall, CJ; Thrasher, AJ; (2001) The embryonic origins of human haematopoiesis. Br J Haematol , 112 (4) 838 - 850.

Martin, F; Toscano, MG; Blundell, M; Frecha, C; Srivastava, GK; Santamaria, M; ... Molina, IJ; + view all (2005) Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences. GENE THER , 12 (8) 715 - 723. 10.1038/sj.gt.3302457.

Mendoza-Naranjo, A; Bouma, G; Pereda, C; Ramirez, M; Webb, KF; Tittarelli, A; ... Salazar-Onfray, F; + view all (2011) Functional Gap Junctions Accumulate at the Immunological Synapse and Contribute to T Cell Activation. J IMMUNOL , 187 (6) 3121 - 3132. 10.4049/jimmunol.1100378.

Metelo, J; Ward, N; Thrasher, AJ; Burns, SO; (2011) Lentivectors Are Efficient Tools to Manipulate the Dendritic Cell Cytoskeleton. CYTOSKELETON , 68 (8) 434 - 445. 10.1002/cm.20521.

Meyer-Bahlburg, A; Becker-Herman, S; Humblet-Baron, S; Khim, S; Weber, M; Bouma, G; ... Rawlings, DJ; + view all (2008) Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis. BLOOD , 112 (10) 4158 - 4169. 10.1182/blood-2008-02-140814.

Mistry, AR; De Alwis, M; Feudner, E; Ali, RR; Thrasher, AJ; (2002) High-titer stocks of adeno-associated virus from replicating amplicons and herpes vectors. Methods Mol Med , 69 445 - 460.

Modlich, U; Knoss, S; Navarro, S; Zychlinski, D; Matzig, T; Brugman, M; ... Baum, C; + view all (2009) Assessing genotoxicity of lenti- and gammaretroviral vectors in vitro. Human Gene Therapy , 20 (11) 1508 - 1508.

Modlich, U; Zychlinski, D; Maetzig, T; Knoess, S; Brugman, MH; Schambach, A; ... Thrasher, AJ; + view all (2009) Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Molecular Therapy , 17 (11) 1919 - 1928. 10.1038/mt.2009.179.

Moeendarbary, E; Valon, L; Fritzsche, M; Harris, AR; Charras, GT; Moulding, DA; ... Mahadevan, L; + view all (2013) The cytoplasm of living cells behaves as a poroelastic material. Nature Materials , 12 (3) 253 - 261. 10.1038/nmat3517.

Moeendarbary, E; Valon, L; Fritzsche, M; Harris, AR; Moulding, DA; Thrasher, AJ; ... Charras, GT; + view all (2013) The cytoplasm of living cells behaves as a poroelastic material. Nat Mater , 12 (3) 253 - 261. 10.1038/nmat3517.

Moeendarbary, E; Valon, L; Harris, A; Moulding, D; Thrasher, A; Stride, E; ... Charras, G; + view all (2012) Cellular Rheology and Hydraulics. BIOPHYSICAL JOURNAL , 102 (3) 563A - 563A.

Mohanty, ST; Cairney, CJ; Chantry, AD; Madan, S; Fernandes, JA; Howe, SJ; ... Bellantuono, I; + view all (2012) A Small Molecule Modulator of Prion Protein Increases Human Mesenchymal Stem Cell Lifespan, Ex Vivo Expansion, and Engraftment to Bone Marrow in NOD/SCID Mice. STEM CELLS , 30 (6) 1134 - 1143. 10.1002/stem.1065.

Montiel-Equihua, CA; Thrasher, AJ; Gaspar, HB; (2012) Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency. Curr Gene Ther , 12 (1) 57 - 65.

Montiel-Equihua, CA; Thrasher, AJ; Gaspar, HB; (2009) Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency. Stem Cells Cloning , 3 1 - 12. Gold open access

Montiel-Equihua, CA; Zhang, L; Carmo, M; Alonso-Ferrero, ME; Blundell, MP; Monkeviciute, A; ... Fairbanks, L; + view all (2012) The Β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity. Molecular Therapy , 20 (7) 1400 - 1409. 10.1038/mt.2012.50.

Montiel-Equihua, CA; Zhang, L; Knight, S; Collins, M; Saadeh, H; Schulz, R; ... Gaspar, HB; + view all (2011) Development of a gene expression system for enhanced erythroid expression. HUMAN GENE THERAPY , 22 (10) A105 - A105.

Montiel-Equihua, CA; Zhang, L; Knight, S; Saadeh, H; Scholz, S; Carmo, M; ... Gaspar, HB; + view all (2012) The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity. Mol Ther , 20 (7) 1400 - 1409. 10.1038/mt.2012.50.

Monypenny, J; Chou, HC; Banon-Rodriguez, I; Thrasher, AJ; Anton, IM; Jones, GE; Calle, Y; (2011) Role of WASP in cell polarity and podosome dynamics of myeloid cells. EUR J CELL BIOL , 90 (2-3) 198 - 204. 10.1016/j.ejcb.2010.05.009.

Moratto, D; Giliani, S; Bonfim, C; Mazzolari, E; Fischer, A; Ochs, HD; ... Notarangelo, LD; + view all (2011) Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. BLOOD , 118 (6) 1675 - 1684. 10.1182/blood-2010-11-319376.

Moreno-Carranza, B; Gentsch, M; Stein, S; Schambach, A; Santilli, G; Rudolf, E; ... Grez, M; + view all (2009) Transgene optimization significantly improves SIN vector titers, gp91(phox) expression and reconstitution of superoxide production in X-CGD cells. GENE THER , 16 (1) 111 - 118. 10.1038/gt.2008.143.

Moreno-Carranza, B; Gentsch, M; Stein, S; Schambach, A; Santilli, G; Rudolf, E; ... Grez, M; + view all (2009) Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther. , 16 (1) 111 - 118.

Morgan, JE; Gross, JG; Pagel, CN; Beauchamp, JR; Fassati, A; Thrasher, AJ; ... Partridge, TA; + view all (2002) Myogenic cell proliferation and generation of a reversible tumorigenic phenotype are triggered by preirradiation of the recipient site. J CELL BIOL , 157 (4) 693 - 702. 10.1083/jcb.200108047.

Morris, E; Gore, M; Baker, A; Thrasher, AJ; (2013) Moving forward on shifting sands: ethical regulation of gene therapy clinical trials in the United kingdom. Mol Ther , 21 (4) 715 - 716. 10.1038/mt.2013.43.

Moschidou, D; Jones, GN; Abdulrazzak, H; Phoolchund, A; Lay, K; Ramasamy, TS; ... Atala, A; + view all (2012) Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Molecular Therapy , 20 (10) 1953 - 1967. 10.1038/mt.2012.117.

Moschidou, D; Mukherjee, S; Blundell, MP; Drews, K; Jones, GN; Abdulrazzak, H; ... Guillot, PV; + view all (2012) Valproic acid confers functional pluripotency to human amniotic fluid stem cells in a transgene-free approach. Mol Ther , 20 (10) 1953 - 1967. 10.1038/mt.2012.117.

Moschidou, D; Mukherjee, S; Blundell, MP; Jones, GN; Atala, AJ; Thrasher, AJ; ... Guillot, PV; + view all (2013) Human mid-trimester amniotic fluid stem cells cultured under embryonic stem cell conditions with valproic acid acquire pluripotent characteristics. Stem Cells Dev , 22 (3) 444 - 458. 10.1089/scd.2012.0267.

Moulding, DA; Blundell, MP; Spiller, DG; White, MRH; Cory, GO; Calle, Y; ... Thrasher, AJ; + view all (2007) Unregulated actin polymerization by WASp causes defects of mitosis and cytokinesis in X-linked neutropenia. J EXP MED , 204 (9) 2213 - 2224. 10.1084/jem.20062324. Gold open access

Moulding, DA; Record, J; Malinova, D; Thrasher, AJ; (2013) Actin cytoskeletal defects in immunodeficiency. Immunological Reviews , 256 (1) 282 - 299. 10.1111/imr.12114. Green and gold open access
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Mukherjee, S; Apolonia, L; Blundell, MP; Bouma, G; Thrasher, AJ; Karwacz, K; ... Collins, MK; + view all (2009) Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy. Journal of Virology , 83 (7) 3094 - 3103. 10.1128/JVI.02519-08.

Mukherjee, S; Santilli, G; Blundell, MP; Navarro, S; Bueren, JA; Thrasher, AJ; (2011) Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using Induced Pluripotent Stem Cells. PLOS ONE , 6 (3) , Article e17565. 10.1371/journal.pone.0017565. Green and gold open access
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Mukherjee, S; Thrasher, AJ; (2014) Gene correction of induced pluripotent stem cells derived from a murine model of X-linked chronic granulomatous disorder. Methods in Molecular Biology , 1114 427 - 440. 10.1007/978-1-62703-761-7-28.

Mukherjee, S; Thrasher, AJ; (2014) Gene correction of induced pluripotent stem cells derived from a murine model of x-linked chronic granulomatous disorder. Methods Mol Biol , 1114 427 - 440. 10.1007/978-1-62703-761-7_28.

Mukherjee, S; Thrasher, AJ; (2013) Gene therapy for PIDs: Progress, pitfalls and prospects. Gene , 525 (2) 174 - 181. 10.1016/j.gene.2013.03.098.

Mukherjee, S; Thrasher, AJ; (2013) Gene therapy for PIDs: progress, pitfalls and prospects. Gene , 525 (2) 174 - 181. 10.1016/j.gene.2013.03.098. Green and gold open access
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Mukherjee, S; Thrasher, AJ; (2011) Progress and Prospects: Advancements in Retroviral Vector Design, Generation, and Application. HUM GENE THER , 22 (10) 1171 - 1174. 10.1089/hum.2011.2523.

Mukherjee, S; Thrasher, AJ; (2011) iPSCs: Unstable Origins? MOL THER , 19 (7) 1188 - 1190. 10.1038/mt.2011.116.

Nabarro, S; Himoudi, N; Papanastasiou, A; Gilmour, K; Gibson, S; Sebire, N; ... Anderson, J; + view all (2005) Coordinated oncogenic transformation and inhibition of host immune responses by the PAX3-FKHR fusion oncoprotein. J EXP MED , 202 (10) 1399 - 1410.

Nabarro, S; Himoudi, N; Papanastasiou, A; Hubank, M; Canderan, G; Anderson, J; ... Sebire, N; + view all (2005) Coordinated oncogenic transformation and inhibition of host immune responses by the PAX3-FKHR fusion oncoprotein. Journal of Experimental Medicine , 202 (10) 1399 - 1410. 10.1084/jem.20050730. Green open access
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Nabarro, S; Kempski, H; Anderson, J; Thrasher, AJ; Amrolia, P; (2003) Generation of immunostimulatory dendritic cells from the malignant clone in patients with juvenile myelomonocytic leukemia [5]. Leukemia , 17 (9) 1910 - 1912. 10.1038/sj.leu.2403059.

Nabarro, S; Thrasher, A; Kempski, H; Anderson, J; (2004) The Generation of Immunostimulatory Dendritic Cells from the Malignant Clone in Patients with Juvenile Myelomonocytic Leukemia. Leukemia , 17 1910 - 1912.

Nibali, L; O'Dea, M; Bouma, G; Parkar, M; Thrasher, AJ; Burns, S; Donos, N; (2010) Genetic Variants Associated With Neutrophil Function in Aggressive Periodontitis and Healthy Controls. J PERIODONTOL , 81 (4) 527 - 534. 10.1902/jop.2010.090543.

Nicklin, SA; Buening, H; Dishart, KL; de Alwis, M; Girod, A; Hacker, U; ... Baker, AH; + view all (2001) Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells. MOL THER , 4 (3) 174 - 181. 10.1006/mthe.2001.0424.

Nowrouzi, A; Cheung, WT; Li, T; Zhang, X; Arens, A; Paruzynski, A; ... Themis, M; + view all (2013) The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis. Mol Ther , 21 (2) 324 - 337. 10.1038/mt.2012.224.

Ochs, HD; Thrasher, AJ; (2006) The Wiskott-Aldrich syndrome. J ALLERGY CLIN IMMUN , 117 (4) 725 - 738. 10.1016/j.jaci.2006.02.005.

Olivier, A; Jeanson-Leh, L; Bouma, G; Compagno, D; Blondeau, J; Seye, K; ... Galy, A; + view all (2006) A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells. MOL THER , 13 (4) 729 - 737. 10.1016/j.ymthe.2005.11.003.

Ott, MG; Hoelzer, D; Schmidt, M; Glimm, H; Fischer, M; Von Kalle, C; ... Thrasher, AJ; + view all (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nature Medicine , 12 (4) 401 - 409. 10.1038/nm1393.

Ozsahin, H; Cavazzana-Calvo, M; Notarangelo, LD; Schulz, A; Thrasher, AJ; Mazzolari, E; ... Fischer, A; + view all (2008) Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. BLOOD , 111 (1) 439 - 445. 10.1182/blood-2007-03-076679.

Pahal, G; Yong, K; Fahey, A; Thomas, NSB; Rodeck, C; Thrasher, A; (1999) Functional characterisation of primitive human haemopoietic cells in foetal blood. BLOOD , 94 (10) 128A - 128A.

Pahal, GS; Jauniaux, E; Kinnon, C; Thrasher, AJ; Rodeck, CH; (2000) Normal development of human fetal hematopoiesis between eight and seventeen weeks' gestation. AM J OBSTET GYNECOL , 183 (4) 1029 - 1034. 10.1067/mob.2000.106976.

Pallant, C; Cathomen, T; Dannemann, N; Quasim, W; Naldini, L; Kinnon, C; ... Thrasher, A; + view all (2011) Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases. HUM GENE THER , 22 (10) A41 - A41.

Paruzynski, A; Arens, A; Gabriel, R; Bartholomae, C; Eckenberg, R; Nowrouzi, A; ... Schmidt, M; + view all (2009) Integration site analysis by non-restrictive (nr) LAM-PCR combined with optimized high-throughput sequencing. HUMAN GENE THERAPY , 20 (11) 1509 - 1510.

Patton, GS; Brouns, G; Thrasher, AJ; McClure, MO; (2001) Transduction of haematopoietic stem cells (HSC's) with human foamy virus (HFV) vectors. AIDS RES HUM RETROV , 17 S58 - S58.

Philpott, NJ; Thrasher, AJ; (2007) Use of nonintegrating lentiviral vectors for gene therapy. HUM GENE THER , 18 (6) 483 - 489. 10.1089/hum.2007.013.

Picard, C; Von Bernuth, H; Ghandil, P; Chrabieh, M; Ku, C-L; Abel, L; ... Bossuyt, X; + view all (2010) Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency. Medicine , 89 (6) 403 - 425. 10.1097/MD.0b013e3181fd8ec3.

Pike-Overzet, K; de Ridder, D; Weerkamp, F; Baert, MRM; Verstegen, MM; Brugman, MH; ... Staal, FJT; + view all (2006) Gene therapy - Is IL2RG oncogenic in T-cell development? NATURE , 443 (7109) E5 - E5. 10.1038/nature05218.

Pike-Overzet, K; de Ridder, D; Weerkamp, F; Baert, MRM; Verstegen, MM; Howe, SJ; ... Staal, FJT; + view all (2006) Ectopic Retroviral Expression of LMO2, but Not IL2R gamma, Blocks Human T-Cell Development from CD34+Progenitor Cells: Implications for Gene Therapy. MOLECULAR THERAPY , 13 S133 - S133.

Pike-Overzet, K; de Ridder, D; Weerkamp, F; Baert, MRM; Verstegen, MMA; Brugman, MH; ... Staal, FJT; + view all (2007) Ectopic retroviral expression of LMO2, but not IL2R gamma, blocks human T-cell development from CD34+cells: implications for leukemogenesis in gene therapy. LEUKEMIA , 21 (4) 754 - 763. 10.1038/sj.leu.2404563.

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Qasim, W; Gaspar, HB; Thrasher, AJ; (2005) T cell suicide gene therapy to aid haematopoietic stem cell transplantation. CURR GENE THER , 5 (1) 121 - 132.

Qasim, W; Gaspar, HB; Thrasher, AJ; (2004) Gene therapy for severe combined immune deficiency. Expert Rev Mol Med , 6 (13) 1 - 15. 10.1017/S1462399404007884.

Qasim, W; Gilmour, KC; Ashton, E; Graham Davies, E; Thrasher, AJ; Jones, A; ... Finn, A; + view all (2001) Protein assays for diagnosis of Wiskott-Aldrich syndrome and X-linked thrombocytopenia. British Journal of Haematology , 113 (4) 861 - 865. 10.1046/j.1365-2141.2001.02832.x.

Qasim, W; Gilmour, KC; Heath, S; Ashton, E; Cranston, T; Thomas, A; ... Gaspar, HB; + view all (2001) Protein assays for diagnosis of Wiskott-Aldrich syndrome and X-linked thrombocytopenia. BRIT J HAEMATOL , 113 (4) 861 - 865.

Qasim, W; King, D; Buddle, J; Verfuerth, S; Kinnon, C; Thrasher, AJ; Gaspar, HB; (2003) The impact of retroviral suicide gene transduction procedures on T cells. BRIT J HAEMATOL , 123 (4) 712 - 719.

Qasim, W; Mackey, T; Sinclair, J; Chatziandreou, I; Kinnon, C; Thrasher, AJ; Gaspar, HB; (2007) Lentiviral vectors for T-cell suicide gene therapy: Preservation of T-cell effector function after cytokine-mediated transduction. MOL THER , 15 (2) 355 - 360. 10.1038/sj.mt.6300042.

Qasim, W; Thrasher, AJ; (2014) Progress and prospects for engineered T cell therapies. Br J Haematol 10.1111/bjh.12981. Green and gold open access
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Qasim, W; Thrasher, AJ; (2006) RISC control for gene therapy. NAT BIOTECHNOL , 24 (6) 661 - 662.

Qasim, W; Thrasher, AJ; Buddle, J; Kinnon, C; Black, ME; Gaspar, HB; (2002) T cell transduction and suicide with an enhanced mutant thymidine kinase. GENE THER , 9 (12) 824 - 827. 10.1038/sj/gt/3301690.

Qasim, W; Thrasher, AJ; Buddle, J; Kinnon, C; Black, ME; Gaspar, HB; (2002) T cell transduction and suicide with an enhanced mutant thymidine kinase. Gene Ther , 9 (12) 824 - 827. 10.1038/sj.gt.3301690.

Qasim, W; Vink, CA; Thrasher, AJ; (2010) Hybrid Lentiviral Vectors. MOL THER , 18 (7) 1263 - 1267. 10.1038/mt.2010.76.

Rahim, AA; Wong, AMS; Howe, SJ; Buckley, SMK; Acosta-Saltos, AD; Elston, KE; ... Raivich, G; + view all (2009) Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors. GENE THER , 16 (4) 509 - 520. 10.1038/gt.2008.186.

Recher, M; Burns, SO; de la Fuente, MA; Volpi, S; Dahlberg, C; Walter, JE; ... Notarangelo, LD; + view all (2012) B cell-intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice. Blood , 119 (12) 2819 - 2828. 10.1182/blood-2011-09-379412.

Reichel, MB; Ali, RR; Thrasher, AJ; Hunt, DM; Bhattacharya, SS; Baker, D; (1998) Immune responses limit adenovirally mediated gene expression in the adult mouse eye. GENE THER , 5 (8) 1038 - 1046.

Reichel, MB; Bainbridge, J; Baker, D; Thrasher, AJ; Bhattacharya, SS; Ali, RR; (2001) An immune response after intraocular administration of an adenoviral vector containing a beta galactosidase reporter gene slows retinal degeneration in the rd mouse. BRIT J OPHTHALMOL , 85 (3) 341 - 344.

Rivat, C; Booth, C; Alonso-Ferrero, M; Blundell, M; Sebire, NJ; Thrasher, AJ; Gaspar, HB; (2013) SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease. Blood , 121 (7) 1073 - 1076. 10.1182/blood-2012-07-445858.

Rivat, C; Santilli, G; Gaspar, HB; Thrasher, AJ; (2012) Gene therapy for primary immunodeficiencies. Hum Gene Ther , 23 (7) 668 - 675. 10.1089/hum.2012.116. Gold open access

Rocca, C; Abdul-Razak, HH; Estevez, FJM; Guenechea, G; Howe, SJ; Holmes, MC; ... Yanez-Munoz, RJ; + view all (2011) ZFN-driven gene targeting to correct mouse Prkdc scid deficiency. HUM GENE THER , 22 (10) A44 - A44.

Sabri, S; Foudi, A; Boukour, S; Franc, B; Charrier, S; Jandrot-Perrus, M; ... Vainchenker, W; + view all (2006) Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment. BLOOD , 108 (1) 134 - 140. 10.1182/blood-2005-03-1219.

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Salzer, U; Birmelin, J; Bacchelli, C; Witte, T; Buchegger-Podbielski, U; Buckridge, S; ... Grimbacher, B; + view all (2007) Sequence analysis of TNFRSF13b, encoding TACI, in patients with systemic lupus erythematosus. J CLIN IMMUNOL , 27 (4) 372 - 377. 10.1007/s10875-007-9094-y.

Salzer, US; Bacchelli, C; Buckridge, S; Pan-Hammar-Strom, Q; Jennings, S; Lougaris, V; ... Thrasher, AJ; + view all (2008) Relevance of biallelic vs monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes. CLINICAL AND EXPERIMENTAL IMMUNOLOGY , 154 207 - 208.

Salzer, US; Bacchelli, C; Buckridge, S; Pan-Hammar-Strom, Q; Jennings, S; Lougaris, V; ... Thrasher, AJ; + view all (2008) Relevance of biallelic vs monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes. CLINICAL AND EXPERIMENTAL IMMUNOLOGY , 154 207 - 208.

Santilli, G; Almarza, E; Brendel, C; Choi, U; Beilin, C; Blundell, MP; ... Thrasher, AJ; + view all (2011) Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells. MOL THER , 19 (1) 122 - 132. 10.1038/mt.2010.226.

Santilli, G; Anderson, J; Thrasher, AJ; Sala, A; (2013) Catechins and antitumor immunity: Not MDSC's cup of tea. Oncoimmunology , 2 (6) e24443 - ?. 10.4161/onci.24443.

Santilli, G; Piotrowska, I; Cantilena, S; Chayka, O; D'Alicarnasso, M; Morgenstern, DA; ... Sala, A; + view all (2013) Polyphenon [corrected] E enhances the antitumor immune response in neuroblastoma by inactivating myeloid suppressor cells. Clin Cancer Res , 19 (5) 1116 - 1125. 10.1158/1078-0432.CCR-12-2528.

Santilli, G; Thornhill, SI; Christine, K; Thrasher, AJ; (2008) Gene therapy of inherited immunodeficiencies. EXPERT OPIN BIOL TH , 8 (4) 397 - 407. 10.1517/14712598.8.4.397.

Sarra, G; Stephens, C; De Alwis, M; Bainbridge, J; Munro, P; Thrasher, AJ; Ali, RR; (2001) Long term restoration of photoreceptor outer segments in the rds mouse after gene replacement therapy. Investigative Ophthalmology and Visual Science , 42 (4) 1878 - ?.

Sarra, GM; Stephens, C; de Alwis, M; Bainbridge, JWB; Smith, AJ; Thrasher, AJ; Ali, RR; (2001) Gene replacement therapy in the retinal degeneration slow (rds)mouse: the effect on retinal degeneration following partial transduction of the retina. HUM MOL GENET , 10 (21) 2353 - 2361.

Sarra, GM; Stephens, C; Schlichtenbrede, FC; Bainbridge, JWB; Thrasher, AJ; Luthert, PJ; Ali, RR; (2002) Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus. VISION RES , 42 (4) 541 - 549.

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Schlichtenbrede, FC; da Cruz, L; Stephens, C; Smith, AJ; Georgiadis, A; Thrasher, AJ; ... Ali, RR; + view all (2003) Long-term evaluation of retinal function in Prph2(Rd2/Rd2) mice following AAV-mediated gene replacement therapy. J GENE MED , 5 (9) 757 - 764. 10.1002/jgm.401.

Schlichtenbrede, FC; MacNeil, A; Bainbridge, JWB; Tschernutter, M; Thrasher, AJ; Smith, AJ; Ali, RR; (2003) Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2(Rd2/Rd2) model of retinal degeneration. GENE THER , 10 (6) 523 - 527. 10.1038/sj.gt.3301929.

Schlichtenbrede, FC; Smith, AJ; Bainbridge, JWB; Thrasher, AJ; Salt, TE; Ali, RR; (2004) Improvement of neuronal visual responses in the superior colliculus in Prph2 (Rd2/Rd2) mice following gene therapy. MOL CELL NEUROSCI , 25 (1) 103 - 110. 10.1016/j.mcn.2003.09.020.

Schwarzwaelder, K; Howe, SJ; Schmidt, M; Brugman, MH; Deichmann, A; Glimm, H; ... von Kalle, C; + view all (2007) Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J CLIN INVEST , 117 (8) 2241 - 2249. 10.1172/JCI31661. Green open access
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Schwickerath, O; Brouns, G; Thrasher, A; Kinnon, C; Roes, J; Casimir, C; (2004) Enhancer-deleted retroviral vectors restore high levels of superoxide generation in a mouse model of CGD. J GENE MED , 6 (6) 603 - 615. 10.1002/jgm.557.

Scobie, L; Hector, RD; Grant, L; Bell, M; Nielsen, AA; Meikle, S; ... Neil, JC; + view all (2009) A Novel Model of SCID-X1 Reconstitution Reveals Predisposition to Retrovirus-induced Lymphoma but No Evidence of gamma C Gene Oncogenicity. MOL THER , 17 (6) 1031 - 1038. 10.1038/mt.2009.59.

Semenova, E; Di, WL; Larcher, F; Ghani, S; Harper, J; Thrasher, A; Qasim, W; (2011) Human involucrin promoter resists spreading methylation and supports compartment specific gene expression in engineered skin grafts. HUM GENE THER , 22 (10) A52 - A52.

Seymour, LW; Thrasher, AJ; (2012) Gene therapy matures in the clinic. NATURE BIOTECHNOLOGY , 30 (7) 588 - 593. 10.1038/nbt.2290.

Shangaris, P; Loukogeorgakis, SP; Blundell, MP; Petra, E; Ramachandra, D; Maghsoudlou, P; ... David, AL; + view all (2014) Congenic Amniotic Fluid Stem Cells Show Stable Long-Term Engraftment in the Haematopoietic System after In Utero Transplantation. REPRODUCTIVE SCIENCES , 21 (3) 83A - 83A.

Sharifi, R; Sinclair, JC; Gilmour, KC; Arkwright, PD; Kinnon, C; Thrasher, AJ; Gaspar, HB; (2004) SAP mediates specific cytotoxic T-cell functions in X-linked lymphoproliferative disease. BLOOD , 103 (10) 3821 - 3827. 10.1182/blood-2003-09-3359.

Siapati, EK; Bonnet, D; Bigger, BW; Themis, M; Miskin, J; Chipchase, D; ... Thrasher, AJ; + view all (2005) Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Molecular Therapy , 12 (3) 537 - 546. 10.1016/j.ymthe.2005.01.022.

Siapati, EK; Kashofer, K; Bonnet, D; Bigger, BW; Themis, M; Thrasher, AJ; (2007) Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells. European Journal of Haematology , 78 (4) 303 - 313. 10.1111/j.1600-0609.2006.00813.x.

Siapati, KE; Barker, S; Kinnon, C; Michalski, A; Anderson, R; Brickell, P; ... Hart, SL; + view all (2003) Improved antitumour immunity in murine neuroblastoma using a combination of IL-2 and IL-12. BRIT J CANCER , 88 (10) 1641 - 1648. 10.1038/sj.bjc.6600928.

Smith, AJ; Schlichtenbrede, FC; Tschernutter, M; Bainbridge, JW; Thrasher, AJ; Ali, RR; (2003) AAV-mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. MOL THER , 8 (2) 188 - 195. 10.1016/S1525-0016(03)00144-8.

Stein, S; Ott, M; Schultze-Strasser, S; Kinner, A; Jauch, A; Burwinkel, B; ... Grez, M; + view all (2009) Gene therapy for chronic granulomatous disease: Ups and downs. Human Gene Therapy , 20 (11) 1440 - 1441.

Stein, S; Ott, MG; Schultze-Strasser, S; Jauch, A; Burwinkel, B; Kinner, A; ... Grez, M; + view all (2010) Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. NAT MED , 16 (2) 198 - U105. 10.1038/nm.2088.

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Thornhill, SI; Grassman, E; Ulaganathan, M; Waddington, SN; Howe, SJ; Van Der Loo, H; ... Thrasher, AJ; + view all (2008) Self-inactivating retroviral vectors for gene therapy of SCID-X1. Human Gene Therapy , 19 (4) O15 - ?.

Thornhill, SI; Howe, SJ; Ulaganathan, M; Gaspar, HB; Kinnon, C; Thrasher, AJ; ... Sebire, NJ; + view all (2008) Self-inactivating gammaretroviral vectors for gene therapy of x-linked severe combined immunodeficiency. Molecular Therapy , 16 (3) 590 - 598. 10.1038/sj.mt.6300393.

Thornhill, SI; Schambach, A; Howe, SJ; Ulaganathan, M; Grassman, E; Williams, D; ... Thrasher, AJ; + view all (2008) Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. MOL THER , 16 (3) 590 - 598. 10.1038/sj.mt.6300393.

Thornhill, SI; Schambach, A; Howe, SJ; Ulaganathan, M; Grassman, E; Williams, D; ... Thrasher, AJ; + view all Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency. Molecular Therapy

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Titman, PS; Morton, L; Skucek, E; Melvin, D; Gaspar, J; Jones, AM; ... Gaspar, HB; + view all (2005) Cognitive and behavioural outcome following haematopoietic cell transplantation for severe congenital immunodeficiencies. Bone Marrow Transplantation , 35 S277 - S278.

Tolar, J; Adair, JE; Antoniou, M; Bartholomae, CC; Becker, PS; Blazar, BR; ... Thrasher, AJ; + view all (2011) Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting. MOL THER , 19 (7) 1193 - 1198. 10.1038/mt.2011.78.

Tolar, J; Becker, PS; Clapp, DW; Hanenberg, H; Diaz de Heredia, C; Kiem, H-P; ... Bueren, J; + view all (2012) Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic. HUMAN GENE THERAPY , 23 (2) 141 - 144. 10.1089/hum.2011.237.

Toscano, MG; Frecha, C; Benabdellah, K; Cobo, M; Blundell, M; Thrasher, AJ; ... Martin, F; + view all (2008) Hematopoietic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of Wiskott-Aldrich syndrome protein. HUM GENE THER , 19 (2) 179 - 197. 10.1089/hum.2007.098.

Tremblay, JP; Xiao, X; Aartsma-Rus, A; Barbas, C; Blau, HM; Bogdanove, AJ; ... Gao, G; + view all (2013) Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Molecular Therapy , 21 (2) 266 - 268. 10.1038/mt.2013.4. Green and gold open access
file

Tschernutter, M; Schlichtenbrede, FC; Howe, S; Balaggan, KS; Munro, PM; Bainbridge, JWB; ... Ali, RR; + view all (2005) Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. GENE THER , 12 (8) 694 - 701. 10.1038/sj.gt.3302460.

Uduehi, A; Mailhos, C; Truman, H; Thrasher, AJ; Kinnon, C; Hart, SL; (2003) Enhancement of integrin-mediated transfection of haematopoietic cells with a synthetic vector system. BIOTECHNOL APPL BIOC , 38 201 - 209. 10.1042/BA20030062.

Van Der Loo, JCM; Grassman, E; Terwilliger, A; Higashimoto, T; Nordling, DL; Malik, P; ... Reeves, L; + view all (2012) Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection. Gene Therapy , 19 (3) 246 - 254. 10.1038/gt.2011.102.

Van Der Loo, JCM; Grassman, E; Terwilliger, A; Higashimoto, T; Nordling, DL; Malik, P; ... Reeves, L; + view all (2012) Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. Gene Therapy , 19 (8) 872 - 876. 10.1038/gt.2012.37.

van der Loo, JCM; Swaney, WP; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; ... Malik, P; + view all (2012) Scale-up and manufacturing of clinical-grade self-inactivating gamma-retroviral vectors by transient transfection. GENE THERAPY , 19 (3) 246 - 254. 10.1038/gt.2011.102.

van der Loo, JCM; Swaney, WP; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; ... Malik, P; + view all (2012) Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency. GENE THERAPY , 19 (8) 872 - 876. 10.1038/gt.2012.37.

van Til, NP; de Boer, H; Mashamba, N; Wabik, A; Huston, M; Visser, TP; ... Wagemaker, G; + view all (2012) Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene. MOLECULAR THERAPY , 20 (10) 1968 - 1980. 10.1038/mt.2012.110.

van Til, NP; Sarwari, R; Visser, TP; Hauer, J; Lagresle-Peyrou, C; van der Velden, G; ... Wagemaker, G; + view all (2013) Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. J Allergy Clin Immunol 10.1016/j.jaci.2013.10.009.

Van Til, NP; Sarwari, R; Visser, TP; Van Der Velden, G; Verstegen, MMA; Wagemaker, G; ... Poliani, PL; + view all (2014) Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome. Journal of Allergy and Clinical Immunology , 133 (4) 1116 - 1123. 10.1016/j.jaci.2013.10.009.

Vink, CA; Gaspar, HB; Gabriel, R; Schmidt, M; McIvor, RS; Thrasher, AJ; Qasim, W; (2009) Sleeping Beauty Transposition From Nonintegrating Lentivirus. MOL THER , 17 (7) 1197 - 1204. 10.1038/mt.2009.94. Gold open access

Waddington, SN; Nivsarkar, MS; Mistry, AR; Buckley, SMK; Kemball-Cook, G; Mosley, KL; ... Coutelle, C; + view all (2004) Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. BLOOD , 104 (9) 2714 - 2721. 10.1182/blood-2004-02-0627.

Walshe, D; Gaspar, HB; Thrasher, AJ; Cale, CM; Gilmour, KC; (2009) Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency. J ALLERGY CLIN IMMUN , 123 (2) 505 - 508. 10.1016/j.jaci.2008.11.041.

Ward, NJ; Buckley, SMK; Rahim, AA; McVey, JH; Thrasher, AJ; Waddington, SN; (2011) Optimised vectors for the treatment of haemophilia A. HUM GENE THER , 22 (10) A109 - A109.

Ward, NJ; Buckley, SMK; Waddington, SN; VandenDriessche, T; Chuah, MKL; Nathwani, AC; ... McVey, JH; + view all (2011) Codon optimization of human factor VIII cDNAs leads to high-level expression. BLOOD , 117 (3) 798 - 807. 10.1182/blood-2010-05-282707.

Westerberg, L; Larsson, M; Hardy, SJ; Fernandez, C; Thrasher, AJ; Severinson, E; (2005) Wiskott-Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response. BLOOD , 105 (3) 1144 - 1152. 10.1182/blood-2004-03-1003.

Westerberg, LS; Meelu, P; Baptista, M; Eston, MA; Adamovich, DA; Cotta-de-Almeida, V; ... Snapper, SB; + view all (2010) Activating WASP mutations associated with X-linked neutropenia result in enhanced actin polymerization, altered cytoskeletal responses, and genomic instability in lymphocytes. J EXP MED , 207 (6) 1145 - 1152. 10.1084/jem.20091245.

White, H; Thrasher, A; Veys, P; Kinnon, C; Gaspar, HB; (2000) Intrinsic defects of B cell function in X-linked severe combined immunodeficiency. EUR J IMMUNOL , 30 (3) 732 - 737.

Williams, DA; Thrasher, AJ; (2014) Concise Review: Lessons Learned From Clinical Trials of Gene Therapy in Monogenic Immunodeficiency Diseases. Stem Cells Transl Med 10.5966/sctm.2013-0206.

Williams, DA; Thrasher, AJ; (2011) Out of harm's way. Nature Biotechnology , 29 (1) 41 - 42. 10.1038/nbt.1750.

Williams, DA; Thrasher, AJ; Baum, C; (2010) Transatlantic consortium spotlights need for changes in gene therapy trials. Molecular Therapy , 18 (11) 1892 - 1892. 10.1038/mt.2010.227.

Worth, A; Thrasher, AJ; Gaspar, HB; (2006) Autoimmune lymphoproliferative syndrome: molecular basis of disease and clinical phenotype. BRIT J HAEMATOL , 133 (2) 124 - 140. 10.1111/j.1365-2141.2006.05993.x.

Worth, AJJ; Metelo, J; Bouma, G; Moulding, D; Fritzsche, M; Vernay, B; ... Burns, SO; + view all (2013) Disease-associated missense mutations in the EVH1 domain disrupt intrinsic WASp function causing dysregulated actin dynamics and impaired dendritic cell migration. BLOOD , 121 (1) 72 - 84. 10.1182/blood-2012-01-403857.

Wright, MJ; Wightman, L; Lilley, C; Coffin, R; Marber, MS; Latchman, DS; ... Thrasher, A; + view all (1999) Differences in timecourse of gene expression following direct intramyocardial injection of recombinant adenovirus, adeno-associated virus and herpes simplex virus. Heart , 81 (SUPPL. 1)

Wright, MJ; Wightman, L; Lilley, C; Coffin, R; Marber, MS; Latchman, DS; ... Thrasher, A; + view all (1998) In vivo gene transfer to the adult rabbit myocardium: Systematic comparison of models and vectors. Heart , 79 (SUPPL. 1)

Wright, MJ; Wightman, LML; de Alwis, M; Lilley, C; Stewart, L; Hart, S; ... Marber, MS; + view all (1998) Intracoronary and direct intramyocardial injection gene transfer to the in vivo rabbit heart: A comparison of viral and non-viral vectors. CIRCULATION , 98 (17) 527 - 527.

Wright, MJ; Wightman, LML; Lilley, C; de Alwis, M; Hart, SL; Miller, A; ... Marber, MS; + view all (2001) In vivo myocardial gene transfer: Optimization, evaluation and direct comparison of gene transfer vectors. BASIC RES CARDIOL , 96 (3) 227 - 236.

Wu, Y; Wu, W; Wong, WM; Ward, E; Thrasher, AJ; Goldblatt, D; ... Gustafsson, K; + view all (2009) Human gamma delta T cells: a lymphoid lineage cell capable of. J Immunol , 183 (9) 5622 - 5629.

Wu, Y; Wu, WT; Wong, WM; Ward, E; Thrasher, AJ; Goldblatt, D; ... Gustafsson, K; + view all (2009) Human gamma delta T Cells: A Lymphoid Lineage Cell Capable of Professional Phagocytosis. J IMMUNOL , 183 (9) 5622 - 5629. 10.4049/jimmunol.0901772.

Xue, SA; Gao, L; Hart, D; Gillmore, R; Qasim, W; Thrasher, A; ... Stauss, H; + view all (2005) Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. Blood , 106 (9) 3062 - 3067. 10.1182/blood-2005-01-0146.

Xue, SA; Gao, LQ; Hart, D; Gillmore, R; Qasim, W; Thrasher, A; ... Stauss, H; + view all (2005) Elimination of human leukemia cells in NOD/SCID mice by WT1-TCR gene-transduced human T cells. BLOOD , 106 (9) 3062 - 3067. 10.1182/blood-2005-01-0146.

Yanez-Munoz, RJ; Balaggan, KS; MacNeil, A; Howe, SJ; Schmidt, M; Smith, AJ; ... Thrasher, AJ; + view all (2006) Effective gene therapy with nonintegrating lentiviral vectors. NAT MED , 12 (3) 348 - 353. 10.1038/nm1365.

Yong, KL; Ahmed, F; Ings, SJ; Pizzey, AR; Blundell, MP; Thrasher, AJ; ... Linch, DC; + view all (2004) Impaired bone marrow homing of cytokine-activated CD34 cells in the NOD/SCID model. Blood , 103 (6) 2079 - 2087. 10.1182/blood-2003-06-1770. Green open access
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Yong, KL; Fahey, A; Pahal, G; Linch, DC; Pizzey, A; Thomas, NSB; ... Thrasher, AJ; + view all (2002) Fetal haemopoietic cells display enhanced migration across endothelium. BRIT J HAEMATOL , 116 (2) 392 - 400.

Zanta-Boussif, MA; Charrier, S; Brice-Ouzet, A; Martin, S; Opolon, P; Thrasher, AJ; ... Galy, A; + view all (2009) Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. GENE THER , 16 (5) 605 - 619. 10.1038/gt.2009.3.

Zhan, H; Gilmour, K; Chan, L; Farzaneh, F; McNicol, AM; Xu, JH; ... Qasim, W; + view all (2013) Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene. PLoS One , 8 (10) e77106 - ?. 10.1371/journal.pone.0077106.

Zhang, F; Frost, AR; Blundell, MP; Bales, O; Antoniou, MN; Thrasher, AJ; (2010) A Ubiquitous Chromatin Opening Element (UCOE) Confers Resistance to DNA Methylation-mediated Silencing of Lentiviral Vectors. MOL THER , 18 (9) 1640 - 1649. 10.1038/mt.2010.132.

Zhang, F; Thornhill, SI; Howe, SJ; Ulaganathan, M; Scharnbach, A; Sinclair, J; ... Thrasher, AJ; + view all (2007) Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. BLOOD , 110 (5) 1448 - 1457. 10.1182/blood-2006-12-060814. Gold open access

Zhang, F; Thrasher, A; Antoniou, M; (2011) The lentiviral vector containing UCOE element driving IL2 receptor gene confers a long term immuno-reconstitution in mouse model of SCID-X1. HUM GENE THER , 22 (10) A110 - A110.

Zhang, L; Montiel-Equihua, CA; Thrasher, AJ; Gaspar, HB; (2011) Dr Lin Zhang. HUMAN GENE THERAPY , 22 (10) A53 - A54.

Zhang, L; Thrasher, AJ; Gaspar, HB; (2013) Current progress on gene therapy for primary immunodeficiencies. Gene Ther 10.1038/gt.2013.21.

Zhang, L; Thrasher, AJ; Gaspar, HB; (2013) Current progress on gene therapy for primary immunodeficiencies. Gene Therapy , 20 (10) 963 - 969. 10.1038/gt.2013.21.

Zhang, XL; de Alwis, M; Hart, SL; Fitzke, FW; Inglis, SC; Boursnell, MEG; ... Thrasher, AJ; + view all (1999) High-titer recombinant adeno-associated virus production from replicating amplicons and herpes vectors deleted for glycoprotein H. HUM GENE THER , 10 (15) 2527 - 2537.

Zicha, D; Allen, WE; Brickell, PM; Kinnon, C; Dunn, GA; Jones, GE; Thrasher, AJ; (1998) Chemotaxis of macrophages is abolished in the Wiskott-Aldrich syndrome. BRIT J HAEMATOL , 101 (4) 659 - 665.

Book chapter

Bouma, G; Thrasher, AJ; (2011) Disorders of phagocyte function. In: UNSPECIFIED (263 - 272).

Kinnon, C; Thrasher, AJ; (2002) Gene therapy for single gene disorders. In: Brooks, G, (ed.) Gene Therapy. The use of DNA as a drug. (71 - 85). Pharmaceutical Press: London.

Thrasher, AJ; Candotti, F; (2006) Hematopoietic stem cell gene therapy. In: Jorde, LB and Little, PFR and Dunn, MJ and Subramaniam, S, (eds.) Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. (759 - 771). John Wiley & Sons Ltd.: Chichester.

Thrasher, AJ; Segal, AW; (2002) Chronic granulomatous disease and other phagocytic disorders. In: Encyclopaedia of Life Sciences.

Conference item

Shaw, SW; David, AL; Blundell, M; Howe, S; Pipino, C; Maghsoudlou, P; ... De Coppi, P; + view all (2011) Sheep amniotic fluid derived CD34+stem cells engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation. Presented at: UNSPECIFIED.

Shaw, SWS; David, AL; Blundell, M; Howe, S; Pipino, C; Maghsoudlou, P; ... De Coppi, P; + view all (2012) CD34+cells derived from sheep amniotic fluid could engraft in NOD-SCID gamma mice and in lambs after prenatal autologous transplantation. Presented at: UNSPECIFIED.

Proceedings paper

Abdul-Razak, H; Rocca, C; Howe, S; Alonso-Ferrero, ME; Gabriel, R; Bartholomae, C; ... Yanez-Munoz, R; + view all (2014) Gene editing of mutated Prkdc gene in classical scid mouse. In: HUMAN GENE THERAPY. (pp. A19 - A19).

Abdul-Razak, HH; Rocca, C; Howe, SJ; Estevez, FJM; Gan, CHV; Roberts, A; ... Yanez-Munoz, RJ; + view all (2010) Towards Correction of the scid Mouse by Gene Targeting. In: HUMAN GENE THERAPY. (pp. 521 - 522). MARY ANN LIEBERT INC

Abdul-Razak, HH; Rocca, C; Howe, SJ; Eugenia Alonso-Ferrero, M; Gabriel, R; Bartholomae, CC; ... Yanez-Munoz, RJ; + view all (2013) Rescue of mouse Prkdc scid deficiency by transplantation of gene-repaired haematopoietic stem cells. In: HUMAN GENE THERAPY. (pp. A6 - A7).

Abdul-Razak, HH; Rocca, CJ; Howe, SJ; Alonso-Ferrero, ME; Gabriel, R; Bartholomae, CC; ... Yanez-Munoz, RJ; + view all (2014) Restoration of T-Cell Functionality in Classical Scid Mouse By Gene Editing. In: MOLECULAR THERAPY. (pp. S291 - S292).

Acosta-Saltos, C; Gonitel, R; Acosta-Saltos, A; Chavda, S; Thrasher, A; Anderson, P; Raivich, G; (2012) Lentiviral GMCSF is neuroinflammatory and proregenerative. In: HUMAN GENE THERAPY. (pp. A17 - A18).

Alonso-Ferrero, ME; Langford-Smith, A; Burke, D; Heales, S; Bigger, BW; Thrasher, A; Gaspar, B; (2012) Gene Therapy for Lysosomal Storage Diseases using an enhanced lentiviral vector delivery system. In: HUMAN GENE THERAPY. (pp. A14 - A14).

Alsubki, R; Field, A-C; Hirwarkar, P; Saudemont, A; Stauss, H; Morris, E; ... Qasim, W; + view all (2013) Engineering cord blood T cells for tumour immunotherapy. In: HUMAN GENE THERAPY. (pp. A20 - A20).

Alsubki, R; Qaseem, W; Thrasher, A; Zhan, H; Saudemont, A; Pule, M; ... Scharenberg, A; + view all (2014) Lentiviral Mediated Redirection of Cord Blood T-Cell Specificity. In: MOLECULAR THERAPY. (pp. S165 - S165).

Alzubi, J; Rahman, S; Pallant, C; Mussolino, C; Thrasher, A; Cathomen, T; (2014) Designer Nuclease Mediated Correction of X-SCID in a Humanized Murine ES Cell Model. In: MOLECULAR THERAPY. (pp. S290 - S290).

Apolonia, L; Waddington, S; Fernandes, C; Ward, N; Bouma, G; Thrasher, A; ... Philpott, N; + view all (2007) Long-term expression from non-integrating lentiviral vectors in muscle. In: HUMAN GENE THERAPY. (pp. 1037 - 1037). MARY ANN LIEBERT INC

Apolonia, L; Waddington, S; Thrasher, AJ; Collins, M; Philpott, N; (2006) Development of non-integrating lentiviral vectors. In: BSGT Manchester 2006.

Assari, T; Goldblatt, D; Thrasher, A; (2004) Investigating mechanisms of inflammatory resolution in chronic gramulomatous disease. In: ESID Versailles, October 2004.

Bailey, SI; Howe, S; Schambach, A; Bohne, J; Kinnon, C; Baum, C; Thrasher, AJ; (2006) Investigating novel retroviral vectors for the treatment of SCID-X1. In: BSGT IC 2006.

Bailey, SI; Howe, S; Schambach, A; Bohne, J; Russell, DW; Baum, C; Thrasher, AJ; (2005) Investigating novel retroviral vectors for the treatment of SCID-X1. In: BSGT Manchester 2005.

Bainbridge, J; Sundaram, V; Robbie, S; Smith, A; Stockman, A; Rubin, G; ... Ali, R; + view all (2011) Gene therapy for eye disease. In: JOURNAL OF MEDICAL GENETICS. (pp. S45 - S45). B M J PUBLISHING GROUP

Bardelli, M; Mukherjee, S; Tai, A; Linden, M; Thrasher, A; Henckaerts, E; (2013) Patient-specific induced pluripotent stem (iPS) cells as a model to study feasibility and safety of AAV-mediated targeted gene addition. In: HUMAN GENE THERAPY. (pp. A37 - A37).

Bardelli, M; Zhang, F; Linden, RM; Thrasher, AJ; Henckaerts, E; (2012) AAV-Mediated Targeted Gene Addition in Patient-Specific SCID-X1 Induced Pluripotent Stem Cells. In: MOLECULAR THERAPY. (pp. S152 - S152).

Bartholomae, C; Balaggan, K; Yanez-Munoz, RJ; Howe, SJ; MacNeil, A; Ali, R; ... Schmidt, M; + view all (2007) Nearly random lentiviral integration site patterns observed in postmitotic rodent cells. In: HUMAN GENE THERAPY. (pp. 1024 - 1024). MARY ANN LIEBERT INC

Bartholomae, CC; Deichmann, A; Schmidt, M; Yanez-Munoz, RJ; Howe, SJ; Schmidt, S; ... von Kalle, C; + view all (2006) Non-random integration and clone selection by lentiviral SIN-LTR vectors. In: BLOOD. (pp. 929A - 930A). AMER SOC HEMATOLOGY

Bartholomae, CC; Yanez-Munoz, RJ; Balaggan, KS; Howe, SJ; MacNeil, A; Ali, RR; ... Schmidt, M; + view all (2008) Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. In: BLOOD CELLS MOLECULES AND DISEASES. (pp. 253 - 253). ACADEMIC PRESS INC ELSEVIER SCIENCE

Beilin, C; Choudhuri, K; Bouma, G; Malinova, D; Llodra, J; Stokes, DL; ... Burns, SO; + view all (2013) Dendritic cell-expressed common cytokine receptor g-chain is essential for effective IL-15 transpresentation to CD4+T cells at the immunological synapse. In: IMMUNOLOGY. (pp. 156 - 156).

Beilin, C; Choudhuri, K; Bouma, G; Thrasher, AJ; Dustin, ML; Burns, SO; (2011) Dendritic cell common G-chain cytokine receptor is required for optimal IL-15 transpresentation to CD4+T cells at the immunological synapse. In: IMMUNOLOGY. (pp. 110 - 110).

Binny, CJ; McIntosh, JH; Tuddenham, E; Waddington, S; Buckley, S; Thrasher, A; ... Davidoff, A; + view all (2010) Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System. In: BLOOD. (pp. 114 - 114). AMER SOC HEMATOLOGY

Blundell, M; Ancliff, P; Cory, G; Jones, G; de Botton, S; Vainchanker, W; Thrasher, AJ; (2004) Unregulated activation of the actin cytoskeleton through WASp promoters cell death and genomic instability. In: ESID Versailles, October 2004. (pp. 127 - ?).

Blundell, MP; Charrier, S; Kinnon, C; Galy, A; Thrasher, AJ; (2005) Lentiviral gene therapy for Wiskotot Aldrich Syndrome. In: BSGT Manchester 2005.

Booth, C; Rivat, C; Alonso-Ferrero, M; Blundell, M; Sebire, NJ; Thrasher, AJ; Gaspar, HB; (2013) Lentiviral-vector-mediated gene therapy for X-linked lymphoproliferative disease restores humoral and cellular functions. In: LANCET. (pp. 25 - 25).

Booth, C; Rivat, C; Blundell, M; Alonso-Ferrera, M; Thrasher, A; Gaspar, B; (2012) LENTIVIRAL VECTOR MEDIATED GENE THERAPY FOR X-LINKED LYMPHOPROLIFERATIVE DISEASE RESTORES HUMORAL AND CELLULAR FUNCTIONS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 130 - 130).

Booth, C; Rivat, C; Ferrero, MA; Blundell, M; Sebire, N; Thrasher, A; Gaspar, HB; (2013) Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP) restores humoral and cellular functions. In: HUMAN GENE THERAPY. (pp. A14 - A15).

Bouma, G; Carter, NA; Recher, M; Notarangelo, LD; Burns, SO; Mauri, C; Thrasher, AJ; (2012) DEFICIENCY OF REGULATORY B CELLS IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENT MICE LEADS TO EXACERBATED AUTOIMMUNE ARTHRITIS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 12 - 12).

Bouma, G; Lang, PA; Shaabani, N; Borkens, S; Scheu, S; Booth, S; ... Lang, KS; + view all (2012) REDUCED TYPE I INTERFERON PRODUCTION BY DENDRITIC CELLS AND WEAKENED ANTIVIRAL IMMUNITY IN WISKOTT-ALDRICH SYNDROME PROTEIN DEFICIENCY. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 46 - 46).

Bowna, G; Doffinger, R; Patel, S; Peskett, E; Sinclair, J; Kumararatne, D; ... Burns, S; + view all (2008) Neutrophil dysfunction 2 in IRAK-(4) deficiency. In: CLINICAL AND EXPERIMENTAL IMMUNOLOGY. (pp. 209 - 209). WILEY-BLACKWELL PUBLISHING, INC

Brooke, NJ; Antonious, M; Thrasher, AJ; (2005) To be announced. In: BSGT Manchester 2005.

Buckley, S; Howe, S; Sheard, V; Anson, D; Parsons, D; Thrasher, A; ... McKay, T; + view all (2007) Significant airway transduction after in utero administration of a gp64 pseudotyped lentiviral vector. In: JOURNAL OF GENE MEDICINE. (pp. 526 - 526).

Buckley, SMK; Howe, SJ; Thrasher, A; Baker, AH; Mckay, TR; Coutelle, C; Waddington, S; (2007) The route of luciferin administration is a critical determinant of detection of pulmonary and nasal luciferase expression by in vivo bioimaging. In: HUMAN GENE THERAPY. (pp. 1075 - 1075). MARY ANN LIEBERT INC

Burns, S; Hardy, S; Bouma, G; Klein, N; Thrasher, AJ; (2008) Dendritic cells require CD18 for normal podosome formation. In: CLINICAL AND EXPERIMENTAL IMMUNOLOGY. (pp. 16 - 16). WILEY-BLACKWELL PUBLISHING, INC

Burns, S; Hardy, S; Klein, N; Thrasher, A; (2007) CD18 is required for normal podosome formation during adhesion and migration of human dendritic cells. In: Medical Research Society Clinical Scientists Day February 07.

Burns, S; Zenner, H; Plagnol, V; Curtis, J; Mok, K; Eisenhut, M; ... Nejentsev, S; + view all (2012) EXOME SEQUENCINGIDENTIFIES LRBA GENE DELETION IN A PATIENT PRESENTING WITH AUTOIMMUNITY WITHOUT HYPOGAMMAGLOBULINEMIA. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 398 - 398).

Calle, Y; Burns, S; Thrasher, AJ; Jones, GE; (2006) The leukocyte podosome. In: EUROPEAN JOURNAL OF CELL BIOLOGY. (pp. 151 - 157). ELSEVIER GMBH, URBAN & FISCHER VERLAG

Calle, Y; Jagger, C; Chambers, T; Fuller, K; Blundell, MP; Chow, J; ... Thrasher, A; + view all (2002) WASp deficiency in mice results in abnormal assembly of podosomes in osteoclasts and defects in bone resorption. In: JOURNAL OF BONE AND MINERAL RESEARCH. (pp. S197 - S197). AMER SOC BONE & MINERAL RES

Carbonaro, DA; Jin, X; Geiger, S; Senadheera, S; Montiel-Equihua, C; Gaspar, B; ... Kohn, DB; + view all (2012) Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID). In: MOLECULAR THERAPY. (pp. S162 - S162).

Carmo, M; Arumugam, P; Montiel-Equiha, C; Alonso-Ferrero, M; Schambach, A; Baum, C; ... Gaspar, B; + view all (2012) DEVELOPMENT OF GENE THERAPY FOR HAEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH) DUE TO PERFORIN DEFICIENCY. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 131 - 131).

Carmo, M; Arumugam, P; Montiel-Equihua, CA; Alonso-Ferrero, ME; Schambach, A; Baum, C; ... Gaspar, B; + view all (2013) Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency - investigation of whether expression of perforin in stem cells has a toxic effect. In: HUMAN GENE THERAPY. (pp. A15 - A15).

Carmo, M; Arumugam, P; Montiel-Equihua, CA; Alonso-Ferrero, ME; Schambach, A; Baum, C; ... Gaspar, B; + view all (2012) Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. In: HUMAN GENE THERAPY. (pp. A3 - A3).

Carmo, M; Arumugam, P; Montiel-Equihua, CA; Alonso-Ferrero, ME; Tiwari, S; Schambach, A; ... Gaspar, B; + view all (2012) Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due to Perforin Deficiency. In: MOLECULAR THERAPY. (pp. S125 - S126).

Carmo, M; Arumugam, P; Tiwari, S; Thrasher, A; Risma, K; Malik, P; ... Jordan, M; + view all (2012) GENE THERAPY FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH): FIXING A CRITICAL 'CIRCUIT BREAKER' IN THE IMMUNE SYSTEM. In: BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION. (pp. S201 - S201).

Carmo, M; Booth, C; Montiel-Equihua, CA; Schambach, A; Baum, C; Thrasher, AJ; Gaspar, HB; (2010) Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency. In: HUMAN GENE THERAPY. (pp. 1455 - 1455). MARY ANN LIEBERT INC

Carmo, M; Booth, C; Montiel-Equihua, CA; Schambach, A; Baum, C; Thrasher, AJ; Gaspar, HB; (2010) Development of Gene Therapy for HLH Due to Perforin Deficiency. In: HUMAN GENE THERAPY. (pp. 508 - 509). MARY ANN LIEBERT INC

Carmo, M; Calero-Garcia, M; Thrasher, AJ; Gaspar, HB; (2014) Development of Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH) Due To Perforin Defi ciency - Corrected T Cell Transplant as Control Therapy. In: MOLECULAR THERAPY. (pp. S8 - S9).

Carmo, M; Calero-Garcia, M; Thrasher, AJ; Gaspar, HB; (2014) Development of gene therapy for hemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency - corrected T cell transplant as control therapy. In: HUMAN GENE THERAPY. (pp. A9 - A9).

Chan, E; Mussolino, C; Thrasher, A; Cathomen, T; Towers, G; Qasim, W; (2013) Mimicry of anti-HIV retrotransposition events by TALEN mediated Cyclophilin insertion into the human TRIM5 locus. In: HUMAN GENE THERAPY. (pp. A171 - A171).

Chan, E; Schaller, T; Eddaoudi, A; Zhan, H; Tan, CP; Jacobsen, M; ... Qasim, W; + view all (2012) Lentiviral gene therapy against human immunodeficiency virus type 1, using a novel human TRIM21-cyclophilin A restriction factor. In: Hum Gene Ther. (pp. 1176 - 1185). Green open access
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Chan, E; Schaller, T; Klein, N; Thrasher, A; Towers, G; Qasim, W; (2010) Lentiviral Delivery of TRIM-Cyclophilin Restriction Factors Against HIV-1. In: HUMAN GENE THERAPY. (pp. 502 - 502). MARY ANN LIEBERT INC

Charrier, S; Blundell, M; Cedrone, G; Louache, F; Vainchenker, W; Thrasher, A; Galy, A; (2012) Efficient mobilization and transduction of WASP-deficient haematopoietic cells in a murine model. In: HUMAN GENE THERAPY. (pp. A111 - A111).

Cheung, W; Nowrouzi, A; Coutelle, C; Waddington, S; Bigger, B; Al Alaf, F; ... Themis, M; + view all (2013) The fetal mouse is a model of genotoxicity that can uncover genes involved in liver cancer. In: HUMAN GENE THERAPY. (pp. A15 - A16).

Chick, H; McDonald, RA; Kritz, AB; Kane, NM; Alba, R; Sessa, WC; ... Baker, AH; + view all (2010) Effect of Nogo-B Overexpression in Vascular Smooth Muscle Cell Migration and Proliferation Mediated by Integration-Deficient Lentiviral Vectors. In: HUMAN GENE THERAPY. (pp. 1203 - 1204). MARY ANN LIEBERT INC

Chick, HE; McDonald, RA; Kritz, AB; Kane, NM; Alba, R; Sessa, WC; ... Baker, AH; + view all (2010) Analysing the Effect of Nogo-B Overexpression in Vascular Smooth Muscle Cell Proliferation and Migration Induced by Non-Integrating or Integrating Lentiviral Vectors. In: HUMAN GENE THERAPY. (pp. 519 - 520). MARY ANN LIEBERT INC

Chiew, M-L; Ballard, S; Stauss, H; Thrasher, A; Longhurst, H; Workman, S; (2012) WORLD IMMUNOLOGY DAY 2012 EDUCATIONAL SYMPOSIUM FOR CLINICIANS AND PATIENTS. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 26 - 26).

Choudhuri, K; Beilin, C; Bouma, G; Llodra, J; Malinova, D; Stokes, D; ... Burns, S; + view all (2013) Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the immunological synapse. In: JOURNAL OF IMMUNOLOGY.

Davies, EG; Gilmour, KC; Parsley, K; Curry, J; Sebire, N; Poliani, L; ... Thrasher, AJ; + view all (2012) THYMUS TRANSPLANTATION FOR COMPLETE DIGEORGE SYNDROME: THE LONDON EXPERIENCE. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 12 - 13).

Deichman, A; Schmidt, M; Abina, SHB; Cavazzana-Calvo, M; Schwarzwaelder, K; Howe, SJ; ... von Kalle, CN; + view all (2008) Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies. In: BLOOD CELLS MOLECULES AND DISEASES. (pp. 262 - 262). ACADEMIC PRESS INC ELSEVIER SCIENCE

Di, W; Ghani, S; Almarza, D; Chen, I; Mellerio, J; Bernadis, C; ... Qasim, W; + view all (2013) Production of gene engineered epithelia sheets for first-in-man use. In: JOURNAL OF INVESTIGATIVE DERMATOLOGY. (pp. S129 - S129).

Duncan, G; Thrasher, AJ; Kane, NM; (2013) Validation of haemogenic endothelial cell populations in the aorta gonad mesonephros of the mouse for microRNA analysis. In: HUMAN GENE THERAPY. (pp. A39 - A39).

Farmer, S; Lorain, S; Thrasher, A; Garcia, L; Muntoni, F; Conti, F; (2011) Correction of FKRP function via RNA trans-splicing. In: NEUROMUSCULAR DISORDERS. (pp. 719 - 720). PERGAMON-ELSEVIER SCIENCE LTD

Farmer, S; Lorain, S; Thrasher, A; Garcia, L; Muntoni, F; Conti, F; (2011) Correction of FKRP function via RNA trans-splicing. In: NEUROMUSCULAR DISORDERS. (pp. S8 - S8). PERGAMON-ELSEVIER SCIENCE LTD

Ferreira, S; Kao, V; Waddington, S; Fang, Z; Ward, N; Thrasher, AJ; Antoniou, MN; (2010) Development of In Utero Lentiviral Vector Gene Therapy Approaches for Inherited Diseases. In: HUMAN GENE THERAPY. (pp. 524 - 524). MARY ANN LIEBERT INC

Gabriel, R; Paruzynski, A; Eckenberg, R; Bartholomae, CC; Nowrouzi, A; Wang, W; ... Schmidt, M; + view all (2008) Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy. In: BLOOD. (pp. 819 - 819). AMER SOC HEMATOLOGY

Gaspar, B; Buckland, K; Rivat, C; Himoudi, N; Gilmour, K; Booth, C; ... Thrasher, A; + view all (2014) Immunological and metabolic correction after lentiviral vector mediated haematopoietic stem cell gene therapy for ADA deficiency. In: HUMAN GENE THERAPY. (pp. A4 - A5).

Gaspar, B; Carmo, M; Arumugam, P; Alonso-Ferrero, M; Schambach, A; Baum, C; ... Thrasher, A; + view all (2012) Development of Gene therapy for HLH due to perforin deficiency and for XLP1. In: HUMAN GENE THERAPY. (pp. A9 - A9).

Gaspar, HB; Bjorkegren, E; Parsley, K; Gilmour, K; Fairbanks, L; Zhang, F; ... Thrasher, AJ; + view all (2008) Reconstitution of immune function in adenosine deaminase deficient severe combined immunodeficiency following hematopoietic stem cell gene therapy. In: BLOOD CELLS MOLECULES AND DISEASES. (pp. 266 - 267). ACADEMIC PRESS INC ELSEVIER SCIENCE

Gaspar, HB; Bjorkegren, E; Parsley, K; Gilmour, KC; Sinclair, J; Zhang, F; ... Thrasher, AJ; + view all (2005) Somatic Gene Therapy for ADA-SCID following cessation of PEG-ADA and use of a mild conditioning regime. In: BSGT Manchester 2005.

Gaspar, HB; Bjorkegren, E; Parsley, K; Gilmour, KC; Sinclair, J; Zhang, F; ... Thrasher, AJ; + view all (2005) Somatic gene therapy for ADA-SCID following cessation of PEG-ADA and use of a mild conditioning regime. In:

Gaspar, HB; Buckland, K; Rivat, C; Himoudi, N; Gilmour, K; Booth, C; ... Thrasher, AJ; + view all (2014) Immunological and Metabolic Correction After Lentiviral Vector Mediated Haematopoietic Stem Cell Gene Therapy for ADA Deficiency. In: MOLECULAR THERAPY. (pp. S106 - S106).

Gaspar, HB; Harwood, C; Leigh, I; Thrasher, A; (2004) Chronic cutaneous papillomavirus disease after haematopoietic stem-cell transplantation in patients with severe combined immune deficiency. In: ESID Versailles, October 2004. (pp. 100 - ?).