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Number of items: 66.

Article

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. Green open access
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Arroyo Hornero, R; Georgiadis, C; Hua, P; Trzupek, D; He, L-Z; Qasim, W; Todd, JA; ... Hester, J; + view all (2020) CD70 expression determines the therapeutic efficacy of expanded human regulatory T cells. Communications Biology , 3 , Article 375. 10.1038/s42003-020-1097-8. Green open access
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Bacchelli, C; Moretti, FA; Carmo, M; Adams, S; Stanescu, HC; Pearce, K; Madkaikar, M; ... Gaspar, HB; + view all (2017) Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency. Journal of Allergy and Clinical Immunology , 139 (2) pp. 634-642. 10.1016/j.jaci.2016.05.036. Green open access
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Barzaghi, F; Hernandez, LCA; Neven, B; Ricci, S; Kucuk, ZY; Bleesing, JJ; Nademi, Z; ... Bacchetta, R; + view all (2018) Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. Journal of Allergy and Clinical Immunology , 141 (3) 1036-1049.e5. 10.1016/j.jaci.2017.10.041. Green open access
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Brown, JR; Morfopoulou, S; Hubb, J; Emmett, WA; Ip, W; Shah, D; Brooks, T; ... Breuer, J; + view all (2015) Astrovirus VA1/HMO-C: An Increasingly Recognized Neurotropic Pathogen in Immunocompromised Patients. Clin Infect Dis , 60 (6) 881 - 888. 10.1093/cid/ciu940. Green open access
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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. Green open access
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Chan, E; Towers, GJ; Qasim, W; (2014) Gene Therapy Strategies to Exploit TRIM Derived Restriction Factors against HIV-1. Viruses , 6 (1) 243 - 263. 10.3390/v6010243. Green open access
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Chiesa, Robert; Georgiadis, Christos; Syed, Farhatullah; Zhan, Hong; Etuk, Annie; Gkazi, Soragia Athina; Preece, Roland; ... Base-Edited, CAR T Group; + view all (2023) Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia. New England Journal of Medicine , 389 pp. 899-910. 10.1056/NEJMoa2300709. Green open access
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Cottineau, J; Kottemann, MC; Lach, FP; Kang, YH; Vély, F; Deenick, EK; Lazarov, T; ... Jouanguy, E; + view all (2017) Inherited GINS1 deficiency underlies growth retardation along with neutropenia and NK cell deficiency. Journal of Clinical Investigation , 127 (5) pp. 1991-2006. 10.1172/JCI90727. Green open access
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Delhove, JMKM; Qasim, W; (2017) Genome-Edited T Cell Therapies. Current Stem Cell Reports , 3 (2) pp. 124-136. 10.1007/s40778-017-0077-5. Green open access
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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. Green open access
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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. Green open access
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Dobson, G; Klein, N; Veys, P; Qasim, W; Silva, J; Cheng, IL; Shingadia, D; ... Bamford, A; + view all (2019) Persistence of HIV reservoir following successful haematopoietic stem cell transplant for juvenile myelomonocytic leukaemia in a child with perinatally acquired HIV. Journal of Virus Eradication , 2019 (5.3) e6-e9. Green open access
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Elfeky, R; Lucchini, G; Lum, S-H; Ottaviano, G; Builes, N; Nademi, Z; Battersby, A; ... Veys, P; + view all (2020) New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT. Blood Advances , 4 (11) pp. 2418-2429. 10.1182/bloodadvances.2019001315. Green open access
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Elfeky, R; Shah, RM; Unni, MN; Ottaviano, G; Rao, K; Chiesa, R; Amrolia, P; ... Veys, P; + view all (2019) New graft manipulation strategies improved outcome of mismatched stem cell transplantation in children with primary immunodeficiencies. Journal of Allergy and Clinical Immunology , 144 (1) pp. 280-293. 10.1016/j.jaci.2019.01.030. Green open access
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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. Green open access
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Elfeky, RA; Furtado-Silva, JM; Chiesa, R; Rao, K; Lucchini, G; Amrolia, P; Worth, A; ... Veys, P; + view all (2018) Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency. Journal of Allergy and Clinical Immunology , 141 (6) 2279-2282.e2. 10.1016/j.jaci.2017.10.051. Green open access
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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. Green open access
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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. Green open access
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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. Green open access
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Field, AC; Qasim, W; (2015) Engineered T cell therapies. Expert Rev Mol Med , 17 , Article e19. 10.1017/erm.2015.14. Green open access
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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. Green open access
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Georgiadis, C; Preece, R; Nickolay, L; Etuk, A; Petrova, A; Ladon, D; Danyi, A; ... Qasim, W; + view all (2018) Long Terminal Repeat CRISPR-CAR-Coupled "Universal" T Cells Mediate Potent Anti-leukemic Effects. Molecular Therapy , 26 (5) pp. 1215-1227. 10.1016/j.ymthe.2018.02.025. Green open access
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Georgiadis, C; Qasim, W; (2017) Emerging applications of gene edited T cells for the treatment of leukemia. Expert Review of Hematology , 10 (9) pp. 753-755. 10.1080/17474086.2017.1350575. Green open access
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Georgiadis, C; Rasaiyaah, J; Gkazi, SA; Preece, R; Etuk, A; Christi, A; Qasim, W; (2021) Base-edited CAR T cells for combinational therapy against T cell malignancies. Leukemia 10.1038/s41375-021-01282-6. (In press). Green open access
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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. Green open access
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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. Green open access
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Gregory, LC; Ferreira, CB; Young-Baird, SK; Williams, HJ; Harakalova, M; van Haaften, G; Rahman, SA; ... Dattani, MT; + view all (2019) Impaired EIF2S3 function associated with a novel phenotype of X-linked hypopituitarism with glucose dysregulation. EBioMedicine 10.1016/j.ebiom.2019.03.013. (In press). Green open access
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Hassan, A; Lee, P; Maggina, P; Xu, JH; Moreira, D; Slatter, M; Nademi, Z; ... Qasim, W; + view all (2014) Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency. Journal of Allergy and Clinical Immunology , 133 (6) pp. 1660-1666. 10.1016/j.jaci.2014.02.042. Green open access
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Hiwarkar, P; Hubank, M; Qasim, W; Chiesa, R; Gilmour, KC; Saudemont, A; Amrolia, PJ; (2017) Cord blood transplantation recapitulates fetal ontogeny with a distinct molecular signature that supports CD4+ T-cell reconstitution. Blood Advances , 1 (24) pp. 2206-2216. 10.1182/bloodadvances.2017010827. Green open access
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Hou, TZ; Verma, N; Wanders, J; Kennedy, A; Soskic, B; Janman, D; Halliday, N; ... Sansom, DM; + view all (2017) Identifying functional defects in patients with immune dysregulation due to LRBA and CTLA-4 mutations. Blood , 129 pp. 1458-1468. 10.1182/blood-2016-10-745174. Green open access
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Ip, W; Silva, JMF; Gaspar, H; Mitra, A; Patel, S; Rao, K; Chiesa, R; ... Qasim, W; + view all (2018) Multicenter phase 1/2 application of adenovirus-specific T cells in high-risk pediatric patients after allogeneic stem cell transplantation. Cytotherapy , 20 (6) pp. 830-838. 10.1016/j.jcyt.2018.03.040. Green open access
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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. Green open access
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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. Green open access
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Lorenzini, T; Fliegauf, M; Klammer, N; Frede, N; Proietti, M; Bulashevska, A; Camacho-Ordonez, N; ... Grimbacher, B; + view all (2020) Characterization of the clinical and immunologic phenotype and management of 157 individuals with 56 distinct heterozygous NFKB1 mutations. Journal of Allergy and Clinical Immunology , 146 (4) pp. 901-911. 10.1016/j.jaci.2019.11.051. Green open access
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Mock, U; Nickolay, L; Philip, B; Cheung, GW; Zhan, H; Johnston, IC; Kaiser, AD; ... Qasim, W; + view all (2016) Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy using CliniMACS prodigy. Cytotherapy , 18 (8) pp. 1002-1011. 10.1016/j.jcyt.2016.05.009. Green open access
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Morfopoulou, S; Brown, JR; Davies, EG; Anderson, G; Virasami, A; Qasim, W; Chong, WK; ... Breuer, J; + view all (2016) Human Coronavirus OC43 Associated with Fatal Encephalitis. [Letter]. New England Journal of Medicine , 375 (5) 497-+. 10.1056/NEJMc1509458. Green open access
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Morfopoulou, S; Mee, ET; Connaughton, SM; Brown, JR; Gilmour, K; Chong, WK; Duprex, WP; ... Breuer, J; + view all (2017) Deep sequencing reveals persistence of cell-associated mumps vaccine virus in chronic encephalitis. Acta Neuropathologica , 133 (1) pp. 139-147. 10.1007/s00401-016-1629-y. Green open access
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Naso, G; Gkazi, SA; Georgiadis, C; Jayarajan, V; Jacków, J; Fleck, R; Allison, L; ... Qasim, W; + view all (2023) Cytosine deaminase base editing to restore COL7A1 in dystrophic epidermolysis bullosa human:murine skin model. JID Innovations , Article 100191. 10.1016/j.xjidi.2023.100191. (In press). Green open access
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Ottaviano, G; Baird, S; Bonney, D; Connor, P; Cummins, M; Evans, P; Gibson, B; ... Vora, A; + view all (2021) The role of immunotherapy in relapse/refractory precursor-B acute lymphoblastic leukaemia: real-life UK/Ireland experience in children and young adults. British Journal of Haematology , 192 (2) e42-e44. 10.1111/bjh.17198. Green open access
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Ottaviano, Giorgio; Georgiadis, Christos; Gkazi, Soragia Athina; Syed, Farhatullah; Zhan, Hong; Etuk, Annie; Preece, Roland; ... TT52 CRISPR-CAR, group; + view all (2022) Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia. Science Translational Medicine , 14 (668) , Article eabq3010. 10.1126/scitranslmed.abq3010. Green open access
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Ottaviano, Giorgio; Qasim, Waseem; (2022) Genome-Edited T Cell Therapies. Hematology / Oncology Clinics of North America , 36 (4) pp. 729-744. 10.1016/j.hoc.2022.03.006. Green open access
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Petrova, A; Georgiadis, C; Naso, G; Fleck, R; Allison, L; McGrath, J; Dazzi, F; ... Qasim, W; + view all (2020) Human Mesenchymal Stromal Cells Engineered to Express Collagen VII Can Restore Anchoring Fibrils in Recessive Dystrophic Epidermolysis Bullosa Skin Graft Chimeras. Journal of Investigative Dermatology , 140 (1) 121-131.e6. 10.1016/j.jid.2019.05.031. Green open access
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Preece, R; Georgiadis, C; Gkazi, SA; Etuk, A; Christi, A; Qasim, W; (2020) 'Mini' U6 Pol III promoter exhibits nucleosome redundancy and supports multiplexed coupling of CRISPR/Cas9 effects. Gene Therapy , 27 pp. 451-458. 10.1038/s41434-020-0142-z. Green open access
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Preece, R; Pavesi, A; Gkazi, SA; Stegmann, KA; Georgiadis, C; Tan, ZM; Joey Aw, JY; ... Qasim, W; + view all (2020) CRISPR mediated base conversion allows discriminatory depletion of endogenous T cell receptors for enhanced synthetic immunity. Molecular Therapy: Methods & Clinical Development 10.1016/j.omtm.2020.09.002. (In press). Green open access
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Qasim, Waseem; (2023) Genome edited allogeneic donor 'universal' chimeric antigen receptor T Cells. Blood , 141 (8) pp. 835-845. 10.1182/blood.2022016204. Green open access
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Qasim, Waseem; (2021) Genome editing of therapeutic T cells. Gene and Genome Editing , 2 , Article 100010. 10.1016/j.ggedit.2021.100010. Green open access
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Qasim, W; (2019) Allogeneic CAR T cell therapies for leukemia. American Journal of Hematology , 94 (S1) S50-S54. 10.1002/ajh.25399. Green open access
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Qasim, W; Thrasher, AJ; (2014) Progress and prospects for engineered T cell therapies. Br J Haematol 10.1111/bjh.12981. Green open access
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Qasim, W; Zhan, H; Samarasinghe, S; Adams, S; Amrolia, P; Stafford, S; Butler, K; ... Veys, P; + view all (2017) Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Science Translational Medicine , 9 (374) , Article eaaj2013. 10.1126/scitranslmed.aaj2013. Green open access
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Rao, K; Adams, S; Qasim, W; Allwood, Z; Worth, A; Silva, J; Lucchini, G; ... Amrolia, P; + view all (2016) Effect of stem cell source on long-term chimerism and event-free survival in children with primary immunodeficiency disorders after fludarabine and melphalan conditioning regimen. Journal of Allergy and Clinical Immunology , 138 (4) pp. 1152-1160. 10.1016/j.jaci.2016.01.053. Green open access
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Rasaiyaah, J; Georgiadis, C; Preece, R; Mock, U; Qasim, W; (2018) TCRαβ/CD3 disruption enables CD3-specific antileukemic T cell immunotherapy. JCI Insight , 3 (13) , Article e99442. 10.1172/jci.insight.99442. Green open access
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Rota, IA; Handel, AE; Maio, S; Klein, F; Dhalla, F; Deadman, ME; Cheuk, S; ... Holländer, GA; + view all (2021) FOXN1 forms higher-order nuclear condensates displaced by mutations causing immunodeficiency. Science Advances , 7 (49) , Article eabj9247. 10.1126/sciadv.abj9247. Green open access
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Shahni, R; Cale, CM; Anderson, G; Osellame, LD; Hambleton, S; Jacques, S; Wadatilake, Y; ... Rahman, S; + view all (2015) Signal transducer and activator of transcription 2 deficiency is a novel disorder of mitochondrial fission. Brain , 138 (10) pp. 2834-2846. 10.1093/brain/awv182. Green open access
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Slatter, MA; Rao, K; Abd Hamid, IJ; Nademi, Z; Chiesa, R; Elfeky, R; Pearce, MS; ... Veys, P; + view all (2018) Treosulfan, Fludarabine Conditioning for HSCT in Children with Primary Immunodeficiency: UK Experience. Biology of Blood and Marrow Transplantation , 24 (3) pp. 529-536. 10.1016/j.bbmt.2017.11.009. Green open access
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Thaventhiran, JED; Lango Allen, H; Burren, OS; Rae, W; Greene, D; Staples, E; Zhang, Z; ... Smith, KGC; + view all (2020) Whole-genome sequencing of a sporadic primary immunodeficiency cohort. Nature , 583 pp. 90-95. 10.1038/s41586-020-2265-1. Green open access
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Thrasher, A; (2019) Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa. JCI Insight , 4 (11) , Article e126243. 10.1172/jci.insight.126243. Green open access
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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. Green open access
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Unni, MNM; Elfeky, R; Rao, K; Nademi, Z; Chiesa, R; Amrolia, P; Skinner, R; ... Slatter, MA; + view all (2018) Non-posttransplant lymphoproliferative disorder malignancy after hematopoietic stem cell transplantation in patients with primary immunodeficiency: UK experience. Journal of Allergy and Clinical Immunology , 141 (6) 2319-2321.e1. 10.1016/j.jaci.2018.02.038. Green open access
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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. Green open access
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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. Green open access
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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). Green open access
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Conference item

Morris, EC; Tendeiro-Rego, R; Richardson, R; Fox, TA; Sillito, F; Holler, A; Thomas, S; ... Stauss, H; + view all (2019) A Phase I Study Evaluating the Safety and Persistence of Allorestricted WT1-TCR Gene Modified Autologous T Cells in Patients with High-Risk Myeloid Malignancies Unsuitable for Allogeneic Stem Cell Transplantation. Presented at: 61st ASH Annual Meeting, Orlando, FL, USA. Green open access
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Thesis

Alsubki, RA; (2017) Editing T cell specificity. Doctoral thesis , UCL (University College London). Green open access
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Georgiadis, C; (2016) Development of Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa. Doctoral thesis , UCL (University College London). Green open access
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Ip, WYW; (2016) Paediatric immune reconstitution with adenovirus adoptive immunotherapy post haematopoietic stem cell transplant. Doctoral thesis , UCL (University College London). Green open access
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This list was generated on Sun Apr 21 05:04:10 2024 BST.