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Number of items: 188.

Article

(2001) Cognitive and behavioural abnormalities in adenosine deaminase deficient severe combined immunodeficiency. BONE MARROW TRANSPLANATATION , 27 256 - ?.

(2001) Screening for genetic defects in primary immunodeficiencies. CPD Bulletin Immunology and Allergy , 2 3 - 7.

Adams, S; Veys, P; Hollifield, J; Gaspar, B; (2004) Cell lineage specific chimerism in post-haematopoietic stem cell transplant patients. Bone Marrow Transplantation , 33 S101 - S102.

Adams, SP; Rashid, S; Premachandra, T; Harvey, K; Ifederu, A; Wilson, MC; Gaspar, HB; (2014) Screening of Neonatal UK Dried Blood Spots Using a Duplex TREC Screening Assay. J Clin Immunol , 34 (3) 323 - 330. 10.1007/s10875-014-0007-6.

Al-Herz, W; Bousfiha, A; Casanova, JL; Chatila, T; Conley, ME; Cunningham-Rundles, C; ... Tang, ML; + view all (2014) Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency. Front Immunol , 5 162 - ?. 10.3389/fimmu.2014.00162.

Albuquerque, W; Gaspar, HB; (2004) Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency. J PEDIATR , 144 (2) 278 - 280. 10.1016/j.jpeds.2003.10.055.

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.

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.

Amrolia, P; Gaspar, HB; Hassan, A; Webb, D; Jones, A; Sturt, N; ... Veys, P; + view all (2000) Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood , 96 (4) 1239 - 1246.

Amrolia, PJ; Muccioli-Casadei, G; Huls, H; Adams, S; Durett, A; Gee, A; ... Brenner, MK; + view all (2006) Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation. Blood , 108 (6) 1797 - 1808. 10.1182/blood-2006-02-001909.

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.

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.

Bennour, E; Ferrand, C; Remy-Martin, JP; Certoux, JM; Gorke, S; Qasim, W; ... Robinet, E; + view all (2008) Abnormal expression of only the CD34 part of a transgenic CD34/herpes simplex virus-thymidine kinase fusion protein is associated with ganciclovir resistance. HUM GENE THER , 19 (7) 699 - 709. 10.1089/hum.2007.060.

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.

Booth, C; Algar, VE; Xu-Bayford, J; Fairbanks, L; Owens, C; Gaspar, HB; (2012) Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency. J Clin Immunol , 32 (3) 449 - 453. 10.1007/s10875-012-9658-3.

Booth, C; Gaspar, HB; (2009) Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics , 3 349 -358. 10.2147/BTT.S3103. Green and gold open access
file

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; Gilmour, KC; Veys, P; Gennery, AR; Slatter, MA; Chapel, H; ... Gaspar, HB; + view all (2011) X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. Blood , 117 (1) 53 - 62. 10.1182/blood-2010-06-284935.

Booth, C; Hershfield, M; Notarangelo, L; Buckley, R; Hoenig, M; Mahlaoui, N; ... Gaspar, HB; + view all (2007) Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006). CLIN IMMUNOL , 123 (2) 139 - 147. 10.1016/j.clin.2006.12.009.

Booth, C; Hershfield, M; Notarangelo, L; Buckley, R; Hoenig, M; Mahlaoui, N; ... Gaspar, HB; + view all (2007) Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006). Clin Immunol , 123 (2) 139 - 147. 10.1016/j.clim.2006.12.009.

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.

Brown, L; Xu-Bayford, J; Allwood, Z; Slatter, M; Cant, A; Davies, EG; ... Gaspar, HB; + view all (2011) Neonatal Diagnosis of Severe Combined Immunodeficiency Leads to Significantly Improved Survival Outcome: The Case for Newborn Screening EDITORIAL COMMENT. OBSTET GYNECOL SURV , 66 (7) 398 - 399. 10.1097/OGX.0b013e3182338432.

Brown, L; Xu-Bayford, J; Allwood, Z; Slatter, M; Cant, A; Davies, EG; ... Gaspar, HB; + view all (2011) Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. BLOOD , 117 (11) 3243 - 3246. 10.1182/blood-2010-08-300384.

Buckland, KF; Bobby Gaspar, H; (2014) Gene and cell therapy for children - New medicines, new challenges? Advanced Drug Delivery Reviews , 73 162 - 169. 10.1016/j.addr.2014.02.010.

Buckland, KF; Bobby Gaspar, H; (2014) Gene and cell therapy for children - New medicines, new challenges? Adv Drug Deliv Rev 10.1016/j.addr.2014.02.010.

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.

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.

Chatziandreou, E; Gilmour, KC; McNicol, AM; Costabile, M; Sinclair, J; Cubitt, D; ... Gaspar, HB; + view all (2007) Capture and generation of adenovirus specific T cells for adoptive immunotherapy. BRIT J HAEMATOL , 136 (1) 117 - 126. 10.1111/j.1365-2141.2006.06386.x.

Cohen, J; Gandhi, M; Naik, P; Cubitt, D; Rao, K; Thaker, U; ... Veys, P; + view all (2005) Increased incidence of EBV-related disease following paediatric stem cell transplantation with reduced-intensity conditioning. Br J Haematol , 129 (2) 229 - 239. 10.1111/j.1365-2141.2005.05439.x.

Cohen, JM; Rogers, V; Gaspar, HB; Jones, A; Davies, EG; Rao, KC; ... Veys, P; + view all (2006) Serial transplantation of mismatched donor hematopoietic cells between HLA-identical sibling pairs with congenital immunodeficiency: in vivo tolerance permits rapid immune reconstitution following T-replete transplantation without GVHD in the secondary recipient. BLOOD , 108 (6) 2124 - 2126. 10.1182/blood-2006-03-009712.

Cohen, JM; Sebire, NJ; Harvey, J; Gaspar, HB; Cathy, C; Jones, A; ... Veys, P; + view all (2007) Successful treatment of lymphoproliferative disease complicating primary immunodeficiency/immunodysregulatory disorders with reduced-intensity allogeneic stem-cell transplantation. BLOOD , 110 (6) 2209 - 2214. 10.1182/blood-2006-12-062174.

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.

Cranston, T; Gilmour, K; Loughlin, S; Walshe, D; Gaspar, HB; Lester, T; ... Jones, AM; + view all (2000) Strategy for molecular diagnosis of immunodeficiency at Great Ormond Street Hospital: a multidisciplinary approach. Journal of Medical Genetics , 37 624 - ?.

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.

Eastwood, D; Gilmour, KC; Gaspar, HB; (2004) Molecular diagnosis of congenital immunodeficiency. Methods Mol Med , 91 91 - 108.

Eastwood, D; Gilmour, KC; Nistala, K; Meaney, C; Chapel, H; Sherrell, Z; ... Gaspar, HB; + view all (2004) Prevalence of SAP gene defects in male patients diagnosed with common variable immunodeficiency. CLIN EXP IMMUNOL , 137 (3) 584 - 588. 10.1111/j.1365-2249.2004.02581.x.

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.

Gaspar, HB; (2014) Tearing RAGs apart. Blood , 123 (2) 156 - 157. 10.1182/blood-2013-11-535633.

Gaspar, HB; (2012) Gene therapy for ADA-SCID: defining the factors for successful outcome. Blood , 120 (18) 3628 - 3629. 10.1182/blood-2012-08-446559.

Gaspar, HB; (2011) Induced pluripotent stem cells and primary immunodeficiencies: A new frontier reached, a new world beyond? J ALLERGY CLIN IMMUN , 127 (6) 1408 - 1409. 10.1016/j.jaci.2011.02.038.

Gaspar, HB; (2010) Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency. IMMUNOL ALLERGY CLIN , 30 (2) 221 - +. 10.1016/j.iac.2010.01.002.

Gaspar, HB; Aiuti, A; Porta, F; Candotti, F; Hershfield, MS; Notarangelo, LD; (2009) How I treat ADA deficiency. BLOOD , 114 (17) 3524 - 3532. 10.1182/blood-2009-06-189209. Gold open access

Gaspar, HB; Amrolia, P; Hassan, A; Webb, D; Jones, A; Sturt, N; ... Veys, P; + view all (2002) Non-myeloablative stem cell transplantation for congenital immunodeficiencies. Recent Results Cancer Res , 159 134 - 142.

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; Conley, ME; (2000) Early B cell defects. CLIN EXP IMMUNOL , 119 (3) 383 - 389.

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; 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; Ferrando, M; Caragol, I; Hernandez, M; Bertran, JM; De gracia, X; ... Espanol, T; + view all (2000) Kinase mutant Btk results in atypical X-linked agammaglobulinaemia phenotype. CLIN EXP IMMUNOL , 120 (2) 346 - 350.

Gaspar, HB; Gilmour, KC; Jones, AM; (2001) Severe combined immunodeficiency--molecular pathogenesis and diagnosis. Arch Dis Child , 84 (2) 169 - 173.

Gaspar, HB; Goldblatt, D; (1997) Immunodeficiency syndromes and recurrent infection. Br J Hosp Med , 58 (11) 565 - 568.

Gaspar, HB; Goldblatt, D; (1997) Investigation of the immune system in recurrent infection. Br J Hosp Med , 58 (10) 517 - 520.

Gaspar, HB; Hammarström, L; Mahlaoui, N; Borte, M; Borte, S; (2014) The Case for Mandatory Newborn Screening for Severe Combined Immunodeficiency (SCID). J Clin Immunol 10.1007/s10875-014-0029-0.

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; Kinnon, C; (2001) X-linked agammaglobulinemia. IMMUNOL ALLERGY CLIN , 21 (1) 23 - +.

Gaspar, HB; Lester, T; Levinsky, RJ; Kinnon, C; (1998) Bruton's tyrosine kinase expression and activity in X-linked agammaglobulinaemia (XLA): the use of protein analysis as a diagnostic indicator of XLA. CLIN EXP IMMUNOL , 111 (2) 334 - 338.

Gaspar, HB; Lester, T; Levinsky, RJ; Kinnon, C; (1998) Analysis of Bruton's tyrosine kinase protein expression and activity in patients with X-linked agammaglobulinemia (XLA):the use of protein analysis as a diagnostic indicator of XLA. Clinical and Experimental Immunology , 111 (2) 334 - 338.

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; Qasim, W; Davies, EG; Rao, K; Amrolia, PJ; Veys, P; (2013) How I treat severe combined immunodeficiency. Blood 10.1182/blood-2013-02-380105.

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; 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 - ?.

GASPER, HB; BRADLEY, LAD; KATZ, F; LOVERING, RC; ROIFMAN, CM; MORGAN, G; ... KINNON, C; + view all (1995) MUTATION ANALYSIS IN BRUTONS TYROSINE KINASE, THE X-LINKED AGAMMAGLOBULINEMIA GENE, INCLUDING IDENTIFICATION OF AN INSERTIONAL HOTSPOT. HUM MOL GENET , 4 (4) 755 - 757.

Güngör, T; Teira, P; Slatter, M; Stussi, G; Stepensky, P; Moshous, D; ... on behalf of the Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation,; + view all (2013) Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet 10.1016/S0140-6736(13)62069-3.

GENEVIER, HC; HINSHELWOOD, S; GASPAR, HB; RIGLEY, KP; BROWN, D; SAELAND, S; ... LOVERING, RC; + view all (1994) EXPRESSION OF BRUTONS TYROSINE KINASE PROTEIN WITHIN THE B-CELL LINEAGE. EUR J IMMUNOL , 24 (12) 3100 - 3105.

Gholam, C; Grigoriadou, S; Gilmour, KC; Gaspar, HB; (2011) Familial haemophagocytic lymphohistiocytosis: advances in the genetic basis, diagnosis and management. CLIN EXP IMMUNOL , 163 (3) 271 - 283. 10.1111/j.1365-2249.2010.04302.x.

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.

Gilmour, KC; Cranston, T; Loughlin, S; Gwyther, J; Lester, T; Espanol, T; ... Gaspar, HB; + view all (2001) Rapid protein-based assays for the diagnosis of T-B+ severe combined immunodeficiency. BRIT J HAEMATOL , 112 (3) 671 - 676.

Gilmour, KC; Fujii, H; Cranston, T; Davies, EG; Kinnon, C; Gaspar, HB; (2001) Defective expression of the interleukin-2/interleukin-15 receptor beta subunit leads to a natural killer cell-deficient form of severe combined immunodeficiency. BLOOD , 98 (3) 877 - 879.

Gilmour, KC; Gaspar, HB; (2003) Pathogenesis and diagnosis of X-linked lymphoproliferative disease. EXPERT REV MOL DIAGN , 3 (5) 549 - 561.

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; Notarangelo, LD; Puck, JM; Buckley, RH; Candotti, F; ... Workshop,; + view all (2009) Improving cellular therapy for primary immune deficiency diseases: Recognition, diagnosis, and management. J ALLERGY CLIN IMMUN , 124 (6) 1152 - 1160. 10.1016/j.jaci.2009.10.022.

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.

Guengoer, T; Teira, P; Slatter, M; Stuessi, G; Stepensky, P; Moshous, D; ... Europe, IEWP; + view all (2014) Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. LANCET , 383 (9915) 436 - 448. 10.1016/50140-6736(13)62069-3.

Hassan, A; Booth, C; Brightwell, A; Allwood, Z; Veys, P; Rao, K; ... Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency,; + view all (2012) Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood , 120 (17) 3615 - 3624. 10.1182/blood-2011-12-396879.

Hislop, AD; Palendira, U; Leese, AM; Arkwright, PD; Rohrlich, PS; Tangye, SG; ... Rickinson, AB; + view all (2010) Impaired Epstein-Barr virus-specific CD8(+) T-cell function in X-linked lymphoproliferative disease is restricted to SLAM family-positive B-cell targets. BLOOD , 116 (17) 3249 - 3257. 10.1182/blood-2009-09-238832.

Hiwarkar, P; Gaspar, HB; Gilmour, K; Jagani, M; Chiesa, R; Bennett-Rees, N; ... Qasim, W; + view all (2013) Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients. Bone Marrow Transplant , 48 (6) 803 - 808. 10.1038/bmt.2012.221.

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.

Jones, AM; Gaspar, HB; (2000) Immunogenetics: changing the face of immunodeficiency. J CLIN PATHOL , 53 (1) 60 - 65.

Kampmann, B; Cubitt, D; Walls, T; Naik, P; Depala, M; Samarasinghe, S; ... Veys, P; + view all (2005) Improved outcome for children with disseminated adenoviral infection following allogeneic stem cell transplantation. Br J Haematol , 130 (4) 595 - 603. 10.1111/j.1365-2141.2005.05649.x.

Lankester, AC; Visser, LF; Hartwig, NG; Bredius, RG; Gaspar, HB; van der, BM; ... Egeler, RM; + view all (2005) Allogeneic stem cell transplantation in X-linked lymphoproliferative disease: two cases in one family and review of the literature. BONE MARROW TRANSPLANT , 36 (2) 99 - 105.

Ljungman, P; Bregni, M; Brune, M; Cornelissen, J; de Witte, T; Dini, G; ... European Grp Blood Marrow Transpl,; + view all (2010) Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe 2009. BONE MARROW TRANSPL , 45 (2) 219 - 234. 10.1038/bmt.2009.141.

MacCarthy-Morrogh, L; Gaspar, HB; Wang, YC; Katz, F; Thompson, L; Layton, M; ... Kinnon, C; + view all (1998) Absence of expression of the Wiskott-Aldrich syndrome protein in peripheral blood cells of Wiskott-Aldrich syndrome patients. CLIN IMMUNOL IMMUNOP , 88 (1) 22 - 27.

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.

Nistala, K; Gilmour, KC; Cranston, T; Davies, EG; Goldblatt, D; Gaspar, HB; Jones, AM; (2001) X-Linked lymphoproliferative disease: three atypical cases. CLIN EXP IMMUNOL , 126 (1) 126 - 130.

Nistala, K; Gilmour, KC; Cranston, T; Davies, EG; Goldblatt, D; Gaspar, HB; Jones, AM; (2001) X-linked lymphoproliferative disease: three atypical cases. Clin Exp Immunol , 126 (1) 126 - 130.

Palendira, U; Low, C; Chan, A; Hislop, AD; Ho, E; Phan, TG; ... Tangye, SG; + view all (2011) Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP. PLOS BIOLOGY , 9 (11) , Article e1001187. 10.1371/journal.pbio.1001187. Green and gold open access
file

Pan-Hammarstrom, Q; Salzer, U; Du, L; Bjorkander, J; Cunningham-Rundles, C; Nelson, DL; ... Hammarstrom, L; + view all (2007) Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency. NAT GENET , 39 (4) 429 - 430.

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.

Passweg, JR; Baldomero, H; Bregni, M; Cesaro, S; Dreger, P; Duarte, RF; ... Transpla, EGBM; + view all (2013) Hematopoietic SCT in Europe: data and trends in 2011. Bone Marrow Transplantation , 48 (9) 1161 -1167. 10.1038/bmt.2013.51. Green open access
file

Passweg, JR; Baldomero, H; Peters, C; Gaspar, HB; Cesaro, S; Dreger, P; ... Madrigal, A; + view all (2014) Hematopoietic SCT in Europe: data and trends in 2012 with special consideration of pediatric transplantation. Bone Marrow Transplant 10.1038/bmt.2014.55.

Pike-Overzet, K; Baum, C; Bredius, RG; Cavazzana, M; Driessen, GJ; Fibbe, WE; ... Staal, FJ; + view all (2014) Successful RAG1-SCID gene therapy depends on the level of RAG1 expression. J Allergy Clin Immunol , 134 (1) 242 - 243. 10.1016/j.jaci.2014.04.033.

Plunkett, FJ; Franzese, O; Belaramani, LL; Fletcher, JM; Gilmour, KC; Sharifi, R; ... Akbar, AN; + view all (2005) The impact of telomere erosion on memory CD8(+) T cells in patients with X-linked lymphoproliferative syndrome. MECH AGEING DEV , 126 (8) 855 - 865. 10.1016/j.mad.2005.03.006.

Qasim, W; Chatziandreou, I; Thrasher, A; Gaspar, H; (2004) Lentiviral vector and suicide gene systems for T-cell therapies. BONE MARROW TRANSPLANTATION , 33 S22 - S22.

Qasim, W; Gaspar, HB; Thrasher, AJ; (2014) "Darwinian" tumor-suppression model unsupported in clinical experience. Mol Ther , 22 (9) 1562 - 1563. 10.1038/mt.2014.147.

Qasim, W; Gaspar, HB; Thrasher, AJ; (2009) Progress and prospects: gene therapy for inherited immunodeficiencies. GENE THER , 16 (11) 1285 - 1291. 10.1038/gt.2009.127.

Qasim, W; Gaspar, HB; Thrasher, AJ; (2007) Update on clinical gene therapy in childhood. ARCH DIS CHILD , 92 (11) 1028 - 1031. 10.1136/adc.2006.108787.

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, K; Zhan, H; Derniame, S; McNicol, AM; Ip, W; ... Gaspar, HB; + view all (2013) Interferon-γ capture T cell therapy for persistent Adenoviraemia following allogeneic haematopoietic stem cell transplantation. Br J Haematol , 161 (3) 449 - 452. 10.1111/bjh.12251.

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; 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.

Ramanan, AV; Hussain, K; Hird, M; Gaspar, HB; (2000) Short limbed skeletal dysplasia associated with combined immunodeficiency and congenital subglottic stenosis: a new constellation of features. CLIN DYSMORPHOL , 9 (3) 173 - 176.

Rao, K; Amrolia, PJ; Jones, A; Cale, CM; Naik, P; King, D; ... Veys, PA; + view all (2005) Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood , 105 (2) 879 - 885. 10.1182/blood-2004-03-0960.

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

Rogers, MH; Lwin, R; Fairbanks, L; Gerritsen, B; Gaspar, HB; (2001) Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency. J PEDIATR , 139 (1) 44 - 50. 10.1067/mpd.2001.115023.

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.

Samarasinghe, S; Mancao, C; Pule, M; Nawroly, N; Karlsson, H; Brewin, J; ... Amrolia, PJ; + view all (2010) Functional characterization of alloreactive T cells identifies CD25 and CD71 as optimal targets for a clinically applicable allodepletion strategy. BLOOD , 115 (2) 396 - 407. 10.1182/blood-2009-08-235895.

Sanchez, JJ; Monaghan, G; Borsting, C; Norbury, G; Morling, N; Gaspar, HB; (2006) Carrier Frequency of a Nonsense Mutation in the Adenosine Deaminase (ADA) Gene Implies a High Incidence of ADA-deficient Severe Combined Immunodeficiency (SCID) in Somalia and a Single, Common Haplotype Indicates Common Ancestry. Ann.Hum.Genet.

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
file

Sebire, NJ; Malone, M; Shah, N; Anderson, G; Gaspar, HB; Cubitt, WD; (2004) Pathology of astrovirus associated diarrhoea in a paediatric bone marrow transplant recipient. J CLIN PATHOL , 57 (9) 1001 - 1003. 10.1136/jcp.2004.017178.

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.

Shaw, SWS; Bollini, S; Nader, KA; Gastadello, A; Mehta, V; Filppi, E; ... David, AL; + view all (2011) Autologous Transplantation of Amniotic Fluid-Derived Mesenchymal Stem Cells Into Sheep Fetuses. CELL TRANSPLANT , 20 (7) 1015 - 1031. 10.3727/096368910X543402.

Skucek, E; Butler, S; Gaspar, HB; Titman, P; (2011) Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency. BONE MARROW TRANSPL , 46 (10) 1314 - 1320. 10.1038/bmt.2010.318.

Straathof, KC; Rao, K; Eyrich, M; Hale, G; Bird, P; Berrie, E; ... Amrolia, PJ; + view all (2009) Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study. Lancet , 374 (9693) 912 - 920. 10.1016/S0140-6736(09)60945-4.

Thapar, N; Lindley, KJ; Kiparissi, F; Elawad, MA; Ashworth, M; Veys, P; ... Shah, N; + view all (2008) Treatment of intractable ulcerating enterocolitis of infancy by allogeneic bone marrow transplantation. CLIN GASTROENTEROL H , 6 (2) 248 - 250. 10.1016/j.cgh.2007.11.019.

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

Thrasher, AJ; Gaspar, HB; (2005) Gene therapy for lympho-hematopoietic disorders. Curr Hematol Rep , 4 (4) 305 - 309.

Thrasher, AJ; Gaspar, HB; (2005) Gene therapy in primary immunodeficiencies. Expert Rev Clin Immunol , 1 (2) 239 - 245. 10.1586/1744666X.1.2.239.

Thrasher, AJ; Gaspar, HB; Baum, C; Modlich, U; Schambach, A; Candotti, F; ... Fischer, A; + view all (2006) Gene therapy - X-SCID transgene leukaemogenicity. NATURE , 443 (7109) E5 - E6. 10.1038/nature05219.

Thrasher, AJ; Hacein-Bey-Abina, S; Gaspar, HB; Blanche, S; Davies, EG; Parsley, K; ... Cavazzana-Calvo, M; + view all (2005) Failure of SCID-X1 gene therapy in older patients. Blood , 105 (11) 4255 - 4257. 10.1182/blood-2004-12-4837.

Titman, P; Pink, E; Skucek, E; Melvin, D; Gaspar, J; Jones, A; ... Gaspar, HB; + view all (2006) Cognitive and behavioural outcome following haematopoietic stem cell transplantation for severe congenital immunodeficiency. Bone Marrow Transplantation , 37 S59 - S60.

Titman, P; Pink, E; Skucek, E; O'Hanlon, K; Cole, TJ; Gaspar, J; ... Gaspar, HB; + view all (2008) Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies. BLOOD , 112 (9) 3907 - 3913. 10.1182/blood-2008-04-151332.

Titman, P; Pink, E; Skucek, E; O'Hanlon, K; Cole, TJ; Gaspar, J; ... Gaspar, HB; + view all (2008) Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood , 112 (9) 3907 - 3913. 10.1182/blood-2008-04-151332.

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.

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

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.

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, AP; Hourihane, JOB; Hodges, E; Gaspar, HB; Smith, J; (2005) Multisystem disease in CAEBV infection. Clinical Immunology , 115 S248 - S248.

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.

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, 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.

Book chapter

Kinnon, C; Gaspar, HB; (2002) Genetics of Inherited Immunodeficiency Diseases and Diagnostic Techniques. In: Manual of Clinical Laboratory Immunology. (810 - 812). ASM Press: Washington.

Kinnon, C; Gaspar, HB; (2000) Genetics of inherited immunodeficiency diseases and diagnostic techniques. In: Manual for Clinical Laboratory Immunology.

Conference item

Shaw, SWS; Bollini, S; Nader, KA; Gastadello, A; Mehta, V; Cananzi, M; ... David, AL; + view all (2010) Wide spread and systemic engraftment after in utero autologous transplantation of amniotic fluid mesenchymal stem cells into sheep fetuses. Presented at: UNSPECIFIED.

Shaw, SWS; Bollini, S; Nader, KA; Gastadello, A; Mehta, V; Filppi, E; ... David, AL; + view all (2010) Widespread Engraftment after In Utero Autologous Transplantation of Amniotic Fluid Derived Mesenchymal Stem Cells into Sheep Fetuses. Presented at: 57th Annual Meeting of the Society-for-Gynecologic-Investigation, Orlando, FL.

Proceedings paper

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; 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).

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).

Engelhardt, KR; Gertz, EM; Keles, S; Schaeffer, AA; Ceja, R; Sassi, A; ... Grimbacher, B; + view all (2012) DOCK8 DEFICIENCY AND A DIAGNOSTIC SCORE TO DIFFERENTIATE IT FROM OTHER HYPER-IGE SYNDROMES. In: JOURNAL OF CLINICAL IMMUNOLOGY. (pp. 332 - 333).

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 - ?).

Gaspar, HB; Parsley, K; Howe, S; Gilmour, K; Sinclair, J; Barington, A; ... Thrasher, A; + view all (2004) Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector. In: ESID Versailles, October 2004. (pp. 100 - ?).

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: ASGT St.Louis 2005.

Gilmour K,; Eastwood D,; Nistala C,; Meaney C,; Chapel H,; Sherrell Z,; ... Gaspar HB,; + view all (2004) Prevalence of SAP gene defects in male patients diagnosed with common variable immunodeficiency. In: ESID Versailles, October 2004. (pp. 125 - ?).

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. In: HUMAN GENE THERAPY. (pp. 421 - 421). MARY ANN LIEBERT INC

Howe, SJ; Schmidt, M; Schwarzwaelder, K; Deichmann, A; Glimm, H; Parsley, K; ... Howe, AJ; + view all (2006) Analysis of retroviralintegration sites in a SCID-X1 gene therapy trial. In: BSGT Manchester 2006.

Kampmann, B; Cubitt, D; Naik, P; Depala, M; Hassan, A; Rao, K; ... Veys, P; + view all (2004) Which children die from overwhelming adenoviral infection following haematopoietic stem cell transplantation? In: Bone Marrow Transplantation. (pp. S22 - S22).

Lucas, VS; Gaspar, H; Thrasher, A; Jones, A; Gaspar, J; Roberts, GJ; Davies, EG; (2002) Dental development following conditioned bone marrow transplantation in early infancy for primary immunodeficiency disorders. In: Bone Marrow Transplantation. (pp. 215 - 215).

Montiel-Equihua, CA; Zhang, L; Knight, S; Saadeh, H; Scholz, S; Carmo, M; ... Gaspar, HB; + view all (2012) Development of a gene expression system for enhanced erythroid expression. In: HUMAN GENE THERAPY. (pp. A11 - A11).

Parsley, KL; Gaspar, HB; Howe, S; King, D; Gilmour, KG; Zhang, F; ... Thrasher, AJ; + view all (2005) Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector. In: BSGT Manchester 2005.

Paruzynski, A; Gabriel, R; Eckenberg, R; Bartholomae, CC; Nowrouzi, A; Arens, A; ... Schmidt, M; + view all (2009) Modeling and Increasing the Genome Accessibility of Viral Integration Sites. In: MOLECULAR THERAPY. (pp. S143 - S143). NATURE PUBLISHING GROUP

Rivat, C; Booth, C; Blundell, M; Thrasher, AJ; Gaspar, HB; (2012) Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP1) restores humoral and cellular functions. In: HUMAN GENE THERAPY. (pp. A8 - A8).

Rivat, C; Booth, C; Blundell, M; Thrasher, AJ; Gaspar, HB; (2012) Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions. In: MOLECULAR THERAPY. (pp. S87 - S88).

Schwarzwaelder, K; Schmidt, M; Howe, S; Prinz, C; Wissler, M; Schmidt, S; ... Von Kalle, C; + view all (2004) Sustained polyclonal hematopoietic repopulation after successful SCID-X1 gene therapy by means of a non random integrating pseudotyped gammaretrovector. In: ESGT Finland Nov 2004.

Sebire, NJ; Malone, M; Anderson, G; Shah, N; Gaspar, HB; Cubitt, WD; (2004) "Human intestinal astrovirus infection in an immunocompromised child following bone marrow transplantation". In: (pp. 39A - ?).

Sebire, NJ; Malone, M; Anderson, G; Shah, N; Gaspar, HB; Cubitt, WD; (2004) Human intestinal astrovirus infection in an immunocomprimised child following bone marrow transplantation. In: JOURNAL OF PATHOLOGY. (pp. 39A - 39A). JOHN WILEY & SONS LTD

Straathof, KC; Rao, K; Eyrich, M; Hale, G; Bird, P; Berrie, E; ... Amrolia, P; + view all (2009) A novel antibody-based minimal-intensity conditioning regimen for children with severe organ toxicity or DNA repair disorders. In: Bone Marrow Transplant. (pp. S77 - S78).

Titman, P; Pink, L; Melvin, D; Gaspar, J; Jones, AM; Davies, EG; ... Gaspar, HB; + view all (2004) Cognitive and behavioiural outcome following haematopoietic stem cell transplantation for severe congenital immunodeficiencies. In: ESID Versailles, October 2004. (pp. 167 - ?).

Walshe, D; Gaspar, HB; Thrasher, AJ; Cale, CM; Gilmour, KC; (2010) STAT5 tyrosine phosphorylation for diagnosis and monitoring of patients with severe combined immunodeficiency. In: CLINICAL AND EXPERIMENTAL IMMUNOLOGY. (pp. 2 - 2). WILEY-BLACKWELL PUBLISHING, INC

Weber, MA; Malone, M; Thrasher, A; Gaspar, HB; Sebire, NJ; (2006) Cutaneous graft versus host disease-like histopathological features following gene therapy. In: JOURNAL OF PATHOLOGY. (pp. 20A - 20A). JOHN WILEY & SONS LTD

Zhan, H; Farzaneh, F; Gilmour, K; Chan, L; McNicol, A-M; Xu-Bayford, JH; ... Qasim, W; + view all (2013) First-in-man use of T cells engineered to express a HSVTK-CD34 sort-suicide gene. In: HUMAN GENE THERAPY. (pp. A10 - A10).

This list was generated on Thu Sep 18 04:36:43 2014 BST.