Browse by UCL people
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Number of items: 152.
Article
Abdul-Razak, HH;
Rocca, CJ;
Howe, SJ;
Alonso-Ferrero, ME;
Wang, J;
Gabriel, R;
Bartholomae, CC;
... Yanez-Munoz, RJ; + view all
(2018)
Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.
Scientific Reports
, 8
, Article 8214. 10.1038/s41598-018-26439-9.
|
Abel, U;
Deichmann, A;
Bartholomae, C;
Schwarzwaelder, K;
Glimm, H;
Howe, S;
Thrasher, A;
... 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.
|
Ahmed, F;
Ings, SJ;
Pizzey, AR;
Blundell, MP;
Thrasher, AJ;
Ye, HT;
Fahey, A;
... Yong, KL; + view all
(2004)
Impaired bone marrow homing of cytokine-activated CD34<sup>+</sup> cells in the NOD/SCID model.
Blood
, 103
(6)
pp. 2079-2087.
10.1182/blood-2003-06-1770.
|
Almarza Novoa, E;
Kasbekar, S;
Thrasher, AJ;
Kohn, DB;
Sevilla, J;
Nguyen, T;
Schwartz, JD;
(2018)
Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.
Journal of Allergy and Clinical Immunology: In Practice
, 6
(4)
1418-1420.e10.
10.1016/j.jaip.2017.12.008.
|
Alzubi, J;
Pallant, C;
Mussolino, C;
Howe, SJ;
Thrasher, AJ;
Cathomen, T;
(2017)
Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.
Scientific Reports
, 7
(1)
, Article 12475. 10.1038/s41598-017-12750-4.
|
Arno, G;
Carss, KJ;
Hull, S;
Zihni, C;
Robson, AG;
Fiorentino, A;
Hardcastle, AJ;
... Yu, P; + view all
(2017)
Biallelic Mutation of ARHGEF18, Involved in the Determination of Epithelial Apicobasal Polarity, Causes Adult-Onset Retinal Degeneration.
The American Journal of Human Genetics
, 100
(2)
pp. 334-342.
10.1016/j.ajhg.2016.12.014.
|
Baker, AH;
Ali, RR;
Thrasher, AJ;
(2016)
Impact of BREXIT on UK Gene and Cell Therapy: The Need for Continued Pan-European Collaboration.
Human Gene Therapy
, 27
(9)
pp. 653-655.
10.1089/hum.2016.29033.ahb.
|
Baptista, MA;
Keszei, M;
Oliveira, M;
Sunahara, KK;
Andersson, J;
Dahlberg, CI;
Worth, AJ;
... Westerberg, LS; + view all
(2016)
Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.
Nature Communications
, 7
, Article 12175. 10.1038/ncomms12175.
|
Baruteau, J;
Cunningham, SC;
Yilmaz, BS;
Perocheau, DP;
Eaglestone, S;
Burke, D;
Thrasher, AJ;
... Gissen, P; + view all
(2021)
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Molecular Therapy - Methods & Clinical Development
, 23
pp. 135-146.
10.1016/j.omtm.2021.09.005.
|
Beilin, C;
Choudhuri, K;
Bouma, G;
Malinova, D;
Llodra, J;
Stokes, D;
Shimaoka, M;
... Burns, S; + view all
(2018)
Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the murine immunological synapse [version 1].
Wellcome Open Research
10.12688/wellcomeopenres.14493.1.
|
Beilin, C;
Choudhuri, K;
Bouma, G;
Malinova, D;
Llodra, J;
Stokes, DL;
Shimaoka, M;
... Burns, SO; + view all
(2018)
Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the murine immunological synapse [version 2; referees: 2 approved].
Wellcome Open Research
, 3
, Article 84. 10.12688/wellcomeopenres.14493.2.
|
Biasco, L;
Izotova, N;
Rivat, C;
Ghorashian, S;
Richardson, R;
Guvenel, A;
Hough, R;
... Amrolia, PJ; + view all
(2021)
Clonal expansion of T memory stem cells determines early anti-leukemic responses and long-term CAR T cell persistence in patients.
Nature Cancer
, 2
pp. 629-64.
10.1038/s43018-021-00207-7.
|
Biswas, A;
Shouval, DS;
Griffith, A;
Goettel, JA;
Field, M;
Kang, YH;
Konnikova, L;
... Snapper, SB; + view all
(2018)
WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis.
Nature Communications
, 9
, Article 1779. 10.1038/s41467-018-03670-6.
|
Blanco, E;
Izotova, N;
Booth, C;
Thrasher, AJ;
(2020)
Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.
Frontiers in Immunology
, 11
, Article 608653. 10.3389/fimmu.2020.608653.
|
Booth, C;
Gaspar, HB;
Thrasher, AJ;
(2016)
Treating Immunodeficiency through HSC Gene Therapy.
Trends In Molecular Medicine
, 22
(4)
pp. 317-327.
10.1016/j.molmed.2016.02.002.
|
Bouma, G;
Carter, NA;
Recher, M;
Malinova, D;
Adriani, M;
Notarangelo, LD;
Burns, SO;
... Thrasher, AJ; + view all
(2014)
Exacerbated experimental arthritis in Wiskott-Aldrich syndrome protein deficiency: modulatory role of regulatory B cells.
Eur J Immunol
, 44
(9)
2692 - 2702.
10.1002/eji.201344245.
|
Bovellan, M;
Romeo, Y;
Biro, M;
Boden, A;
Chugh, P;
Yonis, A;
Vaghela, M;
... Charras, G; + view all
(2014)
Cellular control of cortical actin nucleation.
Curr Biol
, 24
(14)
1628 - 1635.
10.1016/j.cub.2014.05.069.
|
Brendel, C;
Rothe, M;
Santilli, G;
Charrier, S;
Stein, S;
Kunkel, H;
Abriss, D;
... Grez, M; + view all
(2018)
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Human Gene Therapy Clinical Development
, 29
(2)
pp. 69-79.
10.1089/humc.2017.245.
|
Burns, SO;
Zarafov, A;
Thrasher, AJ;
(2017)
Primary immunodeficiencies due to abnormalities of the actin cytoskeleton.
Current Opinion in Hematology
, 24
(1)
pp. 16-22.
10.1097/MOH.0000000000000296.
|
Cabanes-Creus, M;
Navarro, RG;
Zhu, E;
Baltazar, G;
Liao, SHY;
Drouyer, M;
Amaya, AK;
... Lisowski, L; + view all
(2022)
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.
Molecular Therapy - Methods and Clinical Development
, 24
pp. 88-101.
10.1016/j.omtm.2021.11.011.
|
Cabanes-Creus, M;
Ginn, SL;
Amaya, AK;
Liao, SHY;
Westhaus, A;
Hallwirth, CV;
Wilmott, P;
... Lisowski, L; + view all
(2019)
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.
Molecular Therapy: Methods & Clinical Development
, 12
pp. 71-84.
10.1016/j.omtm.2018.10.016.
|
Cabanes-Creus, M;
Hallwirth, CV;
Westhaus, A;
Ng, BH;
Liao, SHY;
Zhu, E;
Navarro, RG;
... Lisowski, L; + view all
(2020)
Restoring the natural tropism of AAV2 vectors for human liver.
Science Translational Medicine
, 12
(560)
, Article eaba3312. 10.1126/scitranslmed.aba3312.
|
Cabanes-Creus, M;
Westhaus, A;
Navarro, RG;
Baltazar, G;
Zhu, E;
Amaya, AK;
Liao, SHY;
... Lisowski, L; + view all
(2020)
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.
Methods & Clinical Development
, 17
pp. 1139-1154.
10.1016/j.omtm.2020.05.004.
|
Carmo, M;
Montiel-Equihua, CA;
Alonso-Ferrero, ME;
Blundell, MP;
Thrasher, AJ;
Gaspar, HB;
Risma, KA;
... Baum, C; + view all
(2015)
Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.
Molecular Therapy
, 23
(4)
737 - 745.
10.1038/mt.2014.242.
|
Carss, KJ;
Arno, G;
Erwood, M;
Stephens, J;
Sanchis-Juan, A;
Hull, S;
Megy, K;
... Raymond, FL; + view all
(2017)
Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease.
American Journal of Human Genetics
, 100
(1)
pp. 75-90.
10.1016/j.ajhg.2016.12.003.
|
Chandra, A;
Zhang, F;
Gilmour, KC;
Webster, D;
Plagnol, V;
Kumararatne, DS;
Burns, SO;
... Thrasher, AJ; + view all
(2016)
Common variable immunodeficiency and natural killer cell lymphopenia caused by Ets-binding site mutation in the IL-2 receptor γ (IL2RG) gene promoter.
Journal of Allergy and Clinical Immunology
, 137
(3)
940-942.e4.
10.1016/j.jaci.2015.08.049.
|
Cossu, G;
Birchall, M;
Brown, T;
De Coppi, P;
Culme-Seymour, E;
Gibbon, S;
Hitchcock, J;
... Wilson, JGS; + view all
(2018)
Lancet Commission: Stem Cells and Regenerative Medicine.
The Lancet
, 391
(10123)
pp. 883-910.
10.1016/S0140-6736(17)31366-1.
|
Counsell, JR;
Asgarian, Z;
Meng, J;
Ferrer, V;
Vink, CA;
Howe, SJ;
Waddington, SN;
... Danos, O; + view all
(2017)
Lentiviral vectors can be used for full-length dystrophin gene therapy.
Scientific Reports
, 7
, Article 44775. 10.1038/srep44775.
|
Davies, G;
Cheung, M;
Gilmour, K;
Maimaris, J;
Curry, J;
Furmanski, A;
Sebire, N;
... Thrasher, A; + view all
(2017)
Thymus transplantation for complete DiGeorge syndrome: European experience.
Journal of Allergy and Clinical Immunology
10.1016/j.jaci.2017.03.020.
(In press).
|
Daza-Cajigal, V;
Albuquerque, AS;
Pearson, J;
Hinley, J;
Mason, AS;
Stahlschmidt, J;
Thrasher, AJ;
... Burns, SO; + view all
(2019)
Loss of Janus Associated Kinase 1 Alters Urothelial Cell Function and Facilitates the Development of Bladder Cancer.
Frontiers in Immunology
, 10
, Article 2065. 10.3389/fimmu.2019.02065.
|
Daza-Cajigal, V;
Albuquerque, AS;
Young, DF;
Ciancanelli, MJ;
Moulding, D;
Angulo, I;
Jeanne-Julien, V;
... Burns, SO; + view all
(2022)
Partial human Janus kinase 1 deficiency predominantly impairs responses to interferon gamma and intracellular control of mycobacteria.
Frontiers in Immunology
, 13
, Article 888427. 10.3389/fimmu.2022.888427.
|
Deola, S;
Guerrouahen, BS;
Sidahmed, H;
Al-Mohannadi, A;
Elnaggar, M;
Elsadig, R;
Abdelalim, EM;
... Cugno, C; + view all
(2018)
Tailoring cells for clinical needs: Meeting report from the Advanced Therapy in Healthcare symposium (October 28–29 2017, Doha, Qatar).
Journal of Translational Medicine
, 16
p. 276.
10.1186/s12967-018-1652-y.
|
Descatoire, Marc;
Fritzen, Remi;
Rotman, Samuel;
Kuntzelman, Genevieve;
Leber, Xavier Charles;
Droz-Georget, Stephanie;
Thrasher, Adrian J;
... Candotti, Fabio; + view all
(2022)
Critical role of WASp in germinal center tolerance through regulation of B cell apoptosis and diversification.
Cell Reports
, 38
(10)
, Article 110474. 10.1016/j.celrep.2022.110474.
|
Di, W-L;
Lwin, SM;
Petrova, A;
Bernadis, C;
Syed, F;
Farzaneh, F;
Moulding, DM;
... Qasim, W; + view all
(2019)
Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial.
Human Gene Therapy
, 30
(9)
pp. 1067-1078.
10.1089/hum.2019.049.
|
Domogala, A;
Blundell, M;
Thrasher, A;
Lowdell, MW;
Madrigal, JA;
Saudemont, A;
(2017)
Natural killer cells differentiated in vitro from cord blood CD34(+) cells are more advantageous for use as an immunotherapy than peripheral blood and cord blood natural killer cells.
Cytotherapy
, 19
(6)
pp. 710-720.
10.1016/j.jcyt.2017.03.068.
|
Eichler, F;
Duncan, C;
Musolino, PL;
Orchard, PJ;
De Oliveira, S;
Thrasher, AJ;
Armant, M;
... Williams, DA; + view all
(2017)
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
The New England Journal of Medicine
, 377
pp. 1630-1638.
10.1056/NEJMoa1700554.
|
Eletto, D;
Burns, SO;
Angulo, I;
Plagnol, V;
Gilmour, KC;
Henriquez, F;
Curtis, J;
... Nejentsev, S; + view all
(2016)
Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection.
Nature Communications
, 7
, Article 13992. 10.1038/ncomms13992.
|
Elfeky, RA;
Furtado-Silva, JM;
Chiesa, R;
Rao, K;
Amrolia, P;
Lucchini, G;
Gilmour, K;
... Veys, P; + view all
(2018)
One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years.
[Letter].
Journal of Allergy and Clinical Immunology
, 142
(5)
1654-1656.e7.
10.1016/j.jaci.2018.06.042.
|
Farmery, JHR;
Smith, ML;
Lynch, AG;
Huissoon, A;
Furnell, A;
Mead, A;
Levine, AP;
... Tan, Y; + view all
(2018)
Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data (vol 8, 1300, 2018).
Scientific Reports
, 8
, Article 13376. 10.1038/s41598-018-31524-0.
|
Farmery, JHR;
Smith, ML;
NIHR BioResource - Rare Diseases, .;
Lynch, AG;
Mead, A;
Levine, AP;
Manzur, A;
... Huissoon, A; + view all
(2018)
Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data.
Scientific Reports
, 8
, Article 1300. 10.1038/s41598-017-14403-y.
|
Ferrari, G;
Thrasher, AJ;
Aiuti, A;
(2021)
Gene therapy using haematopoietic stem and progenitor cells.
Nature Reviews Genetics
, 22
(4)
pp. 216-234.
10.1038/s41576-020-00298-5.
|
Ferreira, CB;
Sumner, RP;
Rodriguez-Plata, MT;
Rasaiyaah, J;
Milne, RS;
Thrasher, AJ;
Qasim, W;
(2020)
Lentiviral Vector Production Titer Is Not Limited in HEK293T by Induced Intracellular Innate Immunity.
Molecular Therapy: Methods & Clinical Development
, 17
pp. 209-219.
10.1016/j.omtm.2019.11.021.
|
Field, AC;
Vink, C;
Gabriel, R;
Al-Subki, R;
Schmidt, M;
Goulden, N;
Stauss, H;
... 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.
|
Fox, Thomas;
Bueren, Juan;
Candotti, Fabio;
Fischer, Alain;
Aiuti, Alessandro;
Lankester, Arjan;
AGORA, Initiative;
(2023)
Access to gene therapy for rare diseases when commercialization is not fit for purpose.
Nature Medicine
, 29
pp. 518-519.
10.1038/s41591-023-02208-8.
|
Furmanski, AL;
O'Shaughnessy, RF;
Saldana, JI;
Blundell, MP;
Thrasher, AJ;
Sebire, NJ;
Davies, EG;
(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.
|
García, M;
Bonafont, J;
Martínez-Palacios, J;
Xu, R;
Turchiano, G;
Svensson, S;
Thrasher, AJ;
... Murillas, R; + view all
(2022)
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.
Molecular Therapy - Methods and Clinical Development
, 27
pp. 96-108.
10.1016/j.omtm.2022.09.005.
|
Garcia-Perez, L;
Van Eggermond, M;
Van Roon, L;
Vloemans, SA;
Cordes, M;
Schambach, A;
Rothe, M;
... Pike-Overzet, K; + view all
(2020)
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Molecular Therapy: Methods & Clinical Development
, 17
pp. 666-682.
10.1016/j.omtm.2020.03.016.
|
Georgiadis, A;
Tschernutter, M;
Bainbridge, JWB;
Balaggan, KS;
Mowat, F;
West, EL;
Munro, PMG;
... 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.
|
Georgiadis, C;
Syed, F;
Petrova, A;
Abdul-Wahab, A;
Lwin, SM;
Farzaneh, F;
Chan, L;
... Qasim, W; + view all
(2016)
Lentiviral Engineered Fibroblasts Expressing Codon Optimized COL7A1 Restore Anchoring Fibrils in RDEB.
Journal of Investigative Dermatology
, 136
(1)
pp. 284-292.
10.1038/jid.2015.364.
|
Ghosh, S;
Carmo, M;
Calero-Garcia, M;
Ricciardelli, I;
Bustamante Ogando, JC;
Blundell, MP;
Schambach, A;
... Gaspar, HB; + view all
(2018)
T cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents Haemophagocytic Lymphohistiocytosis manifestations.
Journal of Allergy and Clinical Immunology
, 142
(3)
904-913.e3.
10.1016/j.jaci.2017.11.050.
|
Ginn, SL;
Hallwirth, CV;
Liao, SHY;
Teber, ET;
Arthur, JW;
Wu, J;
Lee, HC;
... Alexander, IE; + view all
(2017)
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Molecular Therapy - Nucleic Acids
, 6
pp. 1-14.
10.1016/j.omtn.2016.11.011.
|
Gorman, KM;
Meyer, E;
Grozeva, D;
Spinelli, E;
McTague, A;
Sanchis-Juan, A;
Carss, KJ;
... Kurian, MA; + view all
(2019)
Bi-allelic Loss-of-Function CACNA1B Mutations in Progressive Epilepsy-Dyskinesia.
American Journal of Human Genetics
, 104
(5)
pp. 948-956.
10.1016/j.ajhg.2019.03.005.
|
Hawkins, KE;
Moschidou, D;
Faccenda, D;
Wruck, W;
Martin-Trujillo, A;
Hau, KL;
Ranzoni, AM;
... Guillot, PV; + view all
(2017)
Human Amniocytes Are Receptive to Chemically Induced Reprogramming to Pluripotency.
Molecular Therapy
, 25
(2)
pp. 427-442.
10.1016/j.ymthe.2016.11.014.
|
Hong, Y;
Casimir, M;
Houghton, BC;
Zhang, F;
Jensen, B;
Omoyinmi, E;
Torrance, R;
... Eleftheriou, D; + view all
(2022)
Lentiviral Mediated ADA2 Gene Transfer Corrects the Defects Associated With Deficiency of Adenosine Deaminase Type 2.
Frontiers in Immunology
, 13
, Article 852830. 10.3389/fimmu.2022.852830.
|
Ito, Y;
Carss, KJ;
Duarte, ST;
Hartley, T;
Keren, B;
Kurian, MA;
Marey, I;
... Revel-Vilk, S; + view all
(2018)
De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures.
American Journal of Human Genetics
, 103
(1)
pp. 144-153.
10.1016/j.ajhg.2018.06.001.
|
Izotova, N;
Rivat, C;
Baricordi, C;
Blanco, E;
Pellin, D;
Watt, E;
Gkazi, AS;
... Biasco, L; + view all
(2021)
Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans.
Nature Communications
, 12
, Article 1622. 10.1038/s41467-021-21834-9.
|
Khan, KN;
El-Asrag, ME;
Ku, CA;
Holder, GE;
McKibbin, M;
Arno, G;
Poulter, JA;
... for NIHR BioResource-Rare Diseases and UK Inherited Retinal Dise, .; + view all
(2017)
Specific Alleles of CLN7/MFSD8, a Protein That Localizes to Photoreceptor Synaptic Terminals, Cause a Spectrum of Nonsyndromic Retinal Dystrophy.
Investigative Ophthalmology & Visual Science
, 58
(7)
pp. 2906-2914.
10.1167/iovs.16-20608.
|
Klatt, D;
Cheng, E;
Hoffmann, D;
Santilli, G;
Thrasher, AJ;
Brendel, C;
Schambach, A;
(2020)
Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells.
Human Gene Therapy
, 31
(3-4)
pp. 199-210.
10.1089/hum.2019.194.
|
Klatt, D;
Cheng, E;
Philipp, F;
Selich, A;
Dahlke, J;
Schmidt, RE;
Schott, JW;
... Schambach, A; + view all
(2019)
Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.
Stem Cell Reports
, 13
(4)
pp. 590-598.
10.1016/j.stemcr.2019.08.008.
|
Kohn, DB;
Booth, C;
Shaw, KL;
Xu-Bayford, J;
Garabedian, E;
Trevisan, V;
Carbonaro-Sarracino, DA;
... Gaspar, HB; + view all
(2021)
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
New England Journal of Medicine
, 384
pp. 2002-2013.
10.1056/NEJMoa2027675.
|
Kohn, DB;
Booth, C;
Kang, EM;
Pai, S-Y;
Shaw, KL;
Santilli, G;
Armant, M;
... the Net4CGD consortium; + view all
(2020)
Lentiviral gene therapy for X-linked chronic granulomatous disease.
Nature Medicine
, 26
pp. 200-206.
10.1038/s41591-019-0735-5.
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Kreins, AY;
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Boycott, K;
... 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.
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Tuijnenburg, P;
Lango Allen, H;
Burns, SO;
Greene, D;
Jansen, MH;
Staples, E;
Stephens, J;
... NIHR-BioResource – Rare Diseases Consortium, .; + view all
(2018)
Loss of function NFKB1 variants are the most common monogenic cause of CVID in Europeans.
Journal of Allergy and Clinical Immunology
, 142
(4)
pp. 1285-1296.
10.1016/j.jaci.2018.01.039.
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Turro, E;
Astle, WJ;
Megy, K;
Gräf, S;
Greene, D;
Shamardina, O;
Allen, HL;
... Ouwehand, WH; + view all
(2020)
Whole-genome sequencing of patients with rare diseases in a national health system.
Nature
, 583
pp. 96-102.
10.1038/s41586-020-2434-2.
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Vaidyanathan, S;
Kerschner, JL;
Paranjapye, A;
Sinha, V;
Lin, B;
Bedrosian, TA;
Thrasher, AJ;
... Porteus, MH; + view all
(2024)
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV.
Molecular Therapy Nucleic Acids
, 35
(1)
, Article 102134. 10.1016/j.omtn.2024.102134.
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Vandrovcova, J;
Salzer, U;
Grimbacher, B;
Wanders, J;
Rao, K;
Thrasher, A;
Burns, S;
... Cooper, N; + view all
(2019)
FAS mutations are an uncommon cause of immune thrombocytopenia in children and adults without additional features of immunodeficiency.
British Journal of Haematology
, 186
(6)
e163-e165.
10.1111/bjh.15979.
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Wei, W;
Tuna, S;
Keogh, MJ;
Smith, KR;
Aitman, TJ;
Beales, PL;
Bennett, DL;
... Chinnery, PF; + view all
(2019)
Germline selection shapes human mitochondrial DNA diversity.
Science
, 364
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, Article eaau6520. 10.1126/science.aau6520.
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Weisser, M;
Demel, UM;
Stein, S;
Chen-Wichmann, L;
Touzot, F;
Santilli, G;
Sujer, S;
... Grez, M; + view all
(2016)
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.
Journal of Allergy and Clinical Immunology
, 138
(1)
pp. 219-228.
10.1016/j.jaci.2015.11.028.
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Westhaus, Adrian;
Cabanes Creus, Marti;
Jonker, Timo;
Sallard, Erwan;
Navarro, Renina Gale;
Zhu, Erhua;
Baltazar, Grober;
... Lisowski, Leszek; + view all
(2022)
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression.
Human Gene Therapy
, 33
(11-12)
pp. 664-682.
10.1089/hum.2021.278.
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Westhaus, A;
Cabanes Creus, M;
Rybicki, A;
Baltazar, G;
Navarro, RG;
Zhu, E;
Drouyer, M;
... Lisowski, L; + view all
(2020)
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction.
Human Gene Therapy
, 31
(9-10)
pp. 575-589.
10.1089/hum.2019.264.
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Whitworth, J;
Smith, PS;
Martin, J-E;
West, H;
Luchetti, A;
Rodger, F;
Clark, G;
... Maher, ER; + view all
(2018)
Comprehensive Cancer-Predisposition Gene Testing in an Adult Multiple Primary Tumor Series Shows a Broad Range of Deleterious Variants and Atypical Tumor Phenotypes.
The American Journal of Human Genetics
, 103
(1)
pp. 3-18.
10.1016/j.ajhg.2018.04.013.
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Zhan, H;
Gilmour, K;
Chan, L;
Farzaneh, F;
McNicol, AM;
Xu, JH;
Adams, S;
... 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)
, Article e77106. 10.1371/journal.pone.0077106.
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Zhang, F;
Santilli, G;
Thrasher, AJ;
(2017)
Characterization of a core region in the A2UCOE that confers effective anti-silencing activity.
Scientific Reports
, 7
, Article 10213. 10.1038/s41598-017-10222-3.
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Zhang, ZY;
Thrasher, AJ;
Zhang, F;
(2019)
Gene therapy and genome editing for primary immunodeficiency diseases.
Genes and Diseases
10.1016/j.gendis.2019.07.007.
(In press).
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Proceedings paper
Chan, E;
Schaller, T;
Eddaoudi, A;
Zhan, H;
Tan, CP;
Jacobsen, M;
Thrasher, AJ;
... 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).
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Conference item
Abdul-Razak, HH;
Rocca, C;
Howe, SJ;
Alonso-Ferrero, ME;
Wang, J;
Gabriel, R;
Bartholomae, C;
... Yanez-Munoz, RJ; + view all
(2017)
Molecular Evidence of Ex Vivo Gene Editing in a Mouse Model of Immunodeficiency.
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Aragao-Filho, WC;
Shejtman, A;
Zinicola, M;
Siler, U;
Reichenbach, J;
Gaspar, HB;
Thrasher, A;
... Condino-Neto, A; + view all
(2017)
Development of a pCCLChim Lentiviral Vector for Gene Therapy of Patients with Chronic Granulomatous Disease (CGD) due to p47-phox Deficiency.
Presented at: Annual Meeting of the American Academy of Allergy, Asthma and Immunology (AAAAI), Atlanta, Georgia, USA.
|
Panchal, N;
Hougton, B;
Diez, B;
Thrasher, AJ;
Gaspar, HB;
Booth, C;
(2017)
T Cell Gene Therapy Corrects Humoral and Cytotoxic Defects in X-Linked Lymphoproliferative Disease (XLP).
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Poletti, V;
Charrier, S;
Corre, G;
Martin, S;
Gjata, B;
Vignaud, A;
Zhang, F;
... Mavilio, F; + view all
(2017)
Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1).
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Romito, M;
Rai, R;
Santilli, G;
Thrasher, AJ;
Cavazza, A;
(2018)
Targeted gene correction for the treatment of severe combined immunodeficiency caused by mutations in the IL7R gene.
Presented at: Conference on Changing the Face of Modern Medicine - Stem Cell and Gene Therapy, Lausanne, Switzerland.
|
Sereni, L;
Castiello, MC;
Anselmo, A;
Marangoni, F;
Di Silvestre, D;
Motta, S;
Inverso, D;
... Villa, A; + view all
(2017)
Lack of WASp Unveils Intrinsic Platelet Defects Sustaining Inflammation and Autoimmunity.
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Shejtman, A;
Aragao Filho, WC;
Weisser, M;
Zinicola, M;
Booth, C;
Burns, SO;
Siler, U;
... Santilli, G; + view all
(2017)
Lentiviral Gene Therapy for p47(phox) Deficient Chronic Granulomatous Disease.
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Six, E;
Lecoules, A;
Caccavelli, L;
Nobles, C;
Male, F;
Magnani, A;
Roudaut, C;
... Cavazzana, M; + view all
(2017)
A Diversity of Human Hematopoietic Differentiation Programs Identified Through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients.
Presented at: 20th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, DC, United States.
|
Poster
Schott, JW;
Leon-Rico, D;
Armenteros-Monterroso, E;
Buckland, KF;
Rivat, C;
Disakou, A;
Pereira, I;
... Thrasher, AJ; + view all
(2018)
Systematic comparison of culture media and transduction enhancers for optimised CD34⁺ cell-based retroviral gene therapy protocols.
Presented at: Changing the Face of Modern Medicine: Stem Cells & Gene Therapy, Lausanne, Switzerland.
|
Thesis
Alsubki, RA;
(2017)
Editing T cell specificity.
Doctoral thesis , UCL (University College London).
|
Georgiadis, C;
(2016)
Development of Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa.
Doctoral thesis , UCL (University College London).
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