Browse by UCL people
Group by: Type | Date
Number of items: 152.
2024
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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2023
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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.
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Sobrino, S;
Magnani, A;
Semeraro, M;
Martignetti, L;
Cortal, A;
Denis, A;
Couzin, C;
... Six, E; + view all
(2023)
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.
Cell Reports Medicine
, 4
(2)
, Article 100919. 10.1016/j.xcrm.2023.100919.
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2022
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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.
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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.
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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.
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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.
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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.
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Magnani, A;
Semeraro, M;
Adam, F;
Booth, C;
Dupré, L;
Morris, EC;
Gabrion, A;
... Cavazzana, M; + view all
(2022)
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome.
nature medicine
, 28
pp. 71-80.
10.1038/s41591-021-01641-x.
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Rae, William;
Sowerby, John M;
Verhoeven, Dorit;
Youssef, Mariam;
Kotagiri, Prasanti;
Savinykh, Natalia;
Coomber, Eve L;
... Smith, Kenneth GC; + view all
(2022)
Immunodeficiency, autoimmunity, and increased risk of B cell malignancy in humans with TRAF3 mutations.
Science Immunology
, 7
(74)
10.1126/sciimmunol.abn3800.
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Senthil, S;
Thrasher, AJ;
Gilmour, KC;
Wright, T;
Wynn, RF;
(2022)
Wiskott Aldrich Syndrome-2 Caused by Novel Wiskott Aldrich Syndrome Protein–Interacting Protein (WIP) Deficiency Is Associated with Juvenile Myelomonocytic Leukaemia — a Case Report.
Journal of Clinical Immunology
, 43
pp. 82-84.
10.1007/s10875-022-01367-6.
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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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2021
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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.
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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.
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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.
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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.
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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.
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Rai, R;
Thrasher, AJ;
Cavazza, A;
(2021)
Gene editing for the treatment of Primary Immunodeficiency Diseases.
Human Gene Therapy
, 32
(1-2)
pp. 43-51.
10.1089/hum.2020.185.
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Rivers, E;
Hong, Y;
Bajaj-Elliott, M;
Worth, A;
Thrasher, AJ;
(2021)
IL-18: a potential inflammation biomarker in Wiskott Aldrich syndrome.
European Journal of Immunology
10.1002/eji.202049024.
(In press).
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Schejtman, A;
Vetharoy, W;
Choi, U;
Rivat, C;
Theobald, N;
Piras, G;
Leon-Rico, D;
... Santilli, G; + view all
(2021)
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Human Gene Therapy
, 32
(17-18)
pp. 949-958.
10.1089/hum.2020.276.
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Schwarzer, A;
Talbot, SR;
Selich, A;
Morgan, M;
Schott, JW;
Dittrich-Breiholz, O;
Bastone, AL;
... Rothe, M; + view all
(2021)
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Molecular Therapy
10.1016/j.ymthe.2021.06.017.
(In press).
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2020
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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.
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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.
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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.
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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.
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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.
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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.
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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;
Junghanns, F;
Mifsud, W;
Somana, K;
Sebire, N;
Rampling, D;
Worth, A;
... Davies, EG; + view all
(2020)
Correction of both immunodeficiency and hypoparathyroidism by thymus transplantation in complete DiGeorge Syndrome.
American Journal of Transplantation
, 20
(5)
pp. 1447-1450.
10.1111/ajt.15668.
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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.
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Molnár, E;
Radwan, N;
Kovács, G;
Andrikovics, H;
Henriquez, F;
Zarafov, A;
Hayman, M;
... Gilmour, K; + view all
(2020)
Key diagnostic markers for Autoimmune Lymphoproliferative Syndrome with molecular genetic diagnosis.
Blood
, 136
(17)
pp. 1933-1945.
10.1182/blood.2020005486.
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Muñoz, P;
Tristán-Manzano, M;
Sánchez-Gilabert, A;
Santilli, G;
Galy, A;
Thrasher, AJ;
Martin, F;
(2020)
WAS-promoter driven lentiviral vectors mimic closely the lop-sided WASP expression during megakaryocytic differentiation: a comparative study.
Molecular Therapy - Methods & Clinical Development
10.1016/j.omtm.2020.09.006.
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Pai, S-Y;
Thrasher, AJ;
(2020)
Gene therapy for X-linked severe combined immunodeficiency: historical outcomes and current status.
Journal of Allergy and Clinical Immunology
, 146
(2)
pp. 258-261.
10.1016/j.jaci.2020.05.055.
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Piras, G;
Montiel-Equihua, C;
Chan, Y-KA;
Wantuch, S;
Stuckey, D;
Burke, D;
Prunty, H;
... Gaspar, HB; + view all
(2020)
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease.
Molecular Therapy - Methods and Clinical Development
, 18
pp. 558-570.
10.1016/j.omtm.2020.07.001.
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Rai, R;
Romito, M;
Rivers, E;
Turchiano, G;
Blattner, G;
Vetharoy, W;
Ladon, D;
... Cavazza, A; + view all
(2020)
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.
Nature Communications
, 11
(1)
, Article 4034. 10.1038/s41467-020-17626-2.
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Rivers, E;
Rai, R;
Lӧtscher, J;
Hollinshead, M;
Markelj, G;
Thaventheran, J;
Worth, AJ;
... Thrasher, AJ; + view all
(2020)
Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cells.
eLife
, 9
, Article e55547. 10.7554/eLife.55547.
(In press).
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Schejtman, A;
Aragão-Filho, WC;
Clare, S;
Zinicola, M;
Weisser, M;
Burns, SO;
Booth, C;
... Santilli, G; + view all
(2020)
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice.
Gene Therapy
10.1038/s41434-020-0164-6.
(In press).
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Six, E;
Guilloux, A;
Denis, A;
Lecoules, A;
Magnani, A;
Vilette, R;
Male, F;
... Bushman, F; + view all
(2020)
Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.
Blood
, 135
(15)
pp. 1219-1231.
10.1182/blood.2019002350.
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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.
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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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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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2019
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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.
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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.
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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.
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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.
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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.
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Loukogeorgakis, SP;
Shangaris, P;
Bertin, E;
Franzin, C;
Piccoli, M;
Pozzobon, M;
Subramaniam, S;
... De Coppi, P; + view all
(2019)
In Utero Transplantation of Expanded Autologous Amniotic Fluid Stem Cells Results in Long-Term Hematopoietic Engraftment.
Stem Cells
, 37
(9)
pp. 1176-1188.
10.1002/stem.3039.
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Perocheau, D;
Cunningham, S;
Lee, J;
Antinao Diaz, J;
Waddington, SN;
Gilmour, K;
Eaglestone, S;
... Baruteau, J; + view all
(2019)
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.
Human Gene Therapy
, 30
(1)
pp. 79-87.
10.1089/hum.2018.098.
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Record, J;
Sendel, A;
Kritikou, JS;
Kuznetsov, NV;
Brauner, H;
He, M;
Nagy, N;
... Westerberg, LS; + view all
(2019)
An intronic deletion in megakaryoblastic leukemia 1 is associated with hyperproliferation of B cells in triplets with Hodgkin lymphoma.
Haematologica
10.3324/haematol.2019.216317.
(In press).
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Rhodes, CJ;
Batai, K;
Bleda, M;
Haimel, M;
Southgate, L;
Germain, M;
Pauciulo, MW;
... Winslow, C; + view all
(2019)
Genetic determinants of risk in pulmonary arterial hypertension: international genome-wide association studies and meta-analysis.
The Lancet Respiratory Medicine
, 7
(3)
pp. 227-238.
10.1016/S2213-2600(18)30409-0.
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Rivers, E;
Worth, A;
Thrasher, AJ;
Burns, SO;
(2019)
How I manage patients with Wiskott Aldrich syndrome.
British Journal of Haematology
, 185
(4)
pp. 647-655.
10.1111/bjh.15831.
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Romito, M;
Rai, R;
Thrasher, A;
Cavazza, A;
(2019)
Genome editing for blood disorders: state of the art and recent advances.
[Review].
Emerging Topics in Life Science
, 3
(3)
289 -299.
10.1042/ETLS20180147.
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Schott, JW;
León-Rico, D;
Ferreira, CB;
Buckland, KF;
Santilli, G;
Armant, MA;
Schambach, A;
... Thrasher, AJ; + view all
(2019)
Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application.
Molecular Therapy - Methods and Clinical Development
, 14
pp. 134-147.
10.1016/j.omtm.2019.05.015.
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Seidel, MG;
Kindle, G;
Gathmann, B;
Quinti, I;
Buckland, M;
Van Montfrans, J;
Scheible, R;
... ESID Registry Working Party and collaborators; + view all
(2019)
The European Society for Immunodeficiencies (ESID) Registry Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity.
The Journal of Allergy and Clinical Immunology: In Practice
, 7
(6)
pp. 1763-1770.
10.1016/j.jaip.2019.02.004.
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Shangaris, P;
Loukogeorgakis, SP;
Subramaniam, S;
Flouri, C;
Jackson, LH;
Wang, W;
Blundell, MP;
... David, AL; + view all
(2019)
Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
[Corrigendum].
Scientific Reports
, 9
, Article 20214. 10.1038/s41598-019-55754-y.
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Shangaris, P;
Loukogeorgakis, S;
Subramaniam, S;
Flouri, C;
Jackson, L;
WANG, W;
Blundell, M;
... David, A; + view all
(2019)
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Scientific Reports
, 9
, Article 11592. 10.1038/s41598-019-48078-4.
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Thomas, DC;
Charbonnier, L-M;
Schejtman, A;
Aldhekri, H;
Coomber, EL;
Dufficy, ER;
Beenken, AE;
... Smith, KGC; + view all
(2019)
Eros Mutations: Decreased Nadph Oxidase Function and Chronic Granulomatous Disease.
Journal of Allergy and Clinical Immunology
, 143
(2)
, Article e1. 10.1016/j.jaci.2018.09.019.
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Thrasher, A;
(2019)
Loss of the interleukin-6 receptor causes immunodeficiency, atopy, and abnormal inflammatory responses.
Journal of Experimental Medicine
, 216
(9)
, Article 1986. 10.1084/jem.20190344.
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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.
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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;
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Germline selection shapes human mitochondrial DNA diversity.
Science
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Gene therapy and genome editing for primary immunodeficiency diseases.
Genes and Diseases
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2018
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Rocca, CJ;
Howe, SJ;
Alonso-Ferrero, ME;
Wang, J;
Gabriel, R;
Bartholomae, CC;
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Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.
Scientific Reports
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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
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Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the murine immunological synapse [version 1].
Wellcome Open Research
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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
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WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis.
Nature Communications
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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
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Lancet Commission: Stem Cells and Regenerative Medicine.
The Lancet
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Tailoring cells for clinical needs: Meeting report from the Advanced Therapy in Healthcare symposium (October 28–29 2017, Doha, Qatar).
Journal of Translational Medicine
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Elfeky, RA;
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(2018)
One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years.
[Letter].
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2018.06.042.
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Huissoon, A;
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Mead, A;
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Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data (vol 8, 1300, 2018).
Scientific Reports
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Smith, ML;
NIHR BioResource - Rare Diseases, .;
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Mead, A;
Levine, AP;
Manzur, A;
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(2018)
Telomerecat: A ploidy-agnostic method for estimating telomere length from whole genome sequencing data.
Scientific Reports
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Ghosh, S;
Carmo, M;
Calero-Garcia, M;
Ricciardelli, I;
Bustamante Ogando, JC;
Blundell, MP;
Schambach, A;
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(2018)
T cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents Haemophagocytic Lymphohistiocytosis manifestations.
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2017.11.050.
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Ito, Y;
Carss, KJ;
Duarte, ST;
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Keren, B;
Kurian, MA;
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(2018)
De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures.
American Journal of Human Genetics
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10.1016/j.ajhg.2018.06.001.
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Geier, CB;
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Lango, HA;
Thwaites, D;
Atschekzei, F;
Brown, M;
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(2018)
Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency.
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2018.02.007.
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Panchal, N;
Houghton, B;
Diez, B;
Ghosh, S;
Ricciardelli, I;
Thrasher, AJ;
Gaspar, HB;
(2018)
Transfer of gene corrected T cells corrects humoral and cytotoxic defects in X-linked lymphoproliferative disease (XLP1).
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2018.02.053.
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Poletti, V;
Charrier, S;
Corre, G;
Gjata, B;
Vignaud, A;
Zhang, F;
Rothe, M;
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(2018)
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Molecular Therapy - Methods and Clinical Development
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10.1016/j.omtm.2018.03.002.
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Rivers, E;
Worth, A;
Thrasher, AJ;
Burns, SO;
(2018)
Bleeding and splenectomy in Wiskott-Aldrich syndrome: A single-centre experience.
[Letter].
The Journal of Allergy and Clinical Immunology: In Practice
10.1016/j.jaip.2018.07.009.
(In press).
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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.
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Sage, E;
Davies, A;
Kolluri, K;
Patrick, S;
Weil, B;
Rego, RVTP;
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(2018)
Targeted stem cells expressing TRAIL as a therapy for lung Cancer TACTICAL: a phase I/II trial.
Lung Cancer
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Schott, JW;
Leon-Rico, D;
Armenteros-Monterroso, E;
Buckland, KF;
Rivat, C;
Disakou, A;
Pereira, I;
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(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.
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Seidel, MG;
Böhm, K;
Dogu, F;
Worth, A;
Thrasher, A;
Florkin, B;
İkincioğulları, A;
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(2018)
Treatment of Severe Forms of LPS-Responsive Beige-like Anchor Protein (LRBA) Deficiency by Allogeneic Hematopoietic Stem Cell Transplantation.
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2017.04.023.
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Castiello, MC;
Marangoni, F;
Anselmo, A;
di Silvestre, D;
Motta, S;
Draghici, E;
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(2018)
Autonomous role of Wiskott-Aldrich Syndrome platelet deficiency in inducing autoimmunity and inflammation.
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2017.12.1000.
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Shangaris, P;
Loukogeorgakis, SP;
Blundell, MP;
Petra, E;
Shaw, SW;
Ramachandra, DL;
Maghsoudlou, P;
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(2018)
Long-Term Hematopoietic Engraftment of Congenic Amniotic Fluid Stem Cells After in Utero Intraperitoneal Transplantation to Immune Competent Mice.
Stem Cells and Development
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10.1089/scd.2017.0116.
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Lango Allen, H;
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Greene, D;
Jansen, MH;
Staples, E;
Stephens, J;
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(2018)
Loss of function NFKB1 variants are the most common monogenic cause of CVID in Europeans.
Journal of Allergy and Clinical Immunology
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10.1016/j.jaci.2018.01.039.
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Whitworth, J;
Smith, PS;
Martin, J-E;
West, H;
Luchetti, A;
Rodger, F;
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(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
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10.1016/j.ajhg.2018.04.013.
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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.
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Alsubki, RA;
(2017)
Editing T cell specificity.
Doctoral thesis , UCL (University College London).
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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
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Aragao-Filho, WC;
Shejtman, A;
Zinicola, M;
Siler, U;
Reichenbach, J;
Gaspar, HB;
Thrasher, A;
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(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.
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Arno, G;
Carss, KJ;
Hull, S;
Zihni, C;
Robson, AG;
Fiorentino, A;
Hardcastle, AJ;
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(2017)
Biallelic Mutation of ARHGEF18, Involved in the Determination of Epithelial Apicobasal Polarity, Causes Adult-Onset Retinal Degeneration.
The American Journal of Human Genetics
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10.1016/j.ajhg.2016.12.014.
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Burns, SO;
Zarafov, A;
Thrasher, AJ;
(2017)
Primary immunodeficiencies due to abnormalities of the actin cytoskeleton.
Current Opinion in Hematology
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10.1097/MOH.0000000000000296.
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Arno, G;
Erwood, M;
Stephens, J;
Sanchis-Juan, A;
Hull, S;
Megy, K;
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Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease.
American Journal of Human Genetics
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10.1016/j.ajhg.2016.12.003.
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Asgarian, Z;
Meng, J;
Ferrer, V;
Vink, CA;
Howe, SJ;
Waddington, SN;
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Lentiviral vectors can be used for full-length dystrophin gene therapy.
Scientific Reports
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Davies, G;
Cheung, M;
Gilmour, K;
Maimaris, J;
Curry, J;
Furmanski, A;
Sebire, N;
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(2017)
Thymus transplantation for complete DiGeorge syndrome: European experience.
Journal of Allergy and Clinical Immunology
10.1016/j.jaci.2017.03.020.
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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
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10.1016/j.jcyt.2017.03.068.
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Duncan, C;
Musolino, PL;
Orchard, PJ;
De Oliveira, S;
Thrasher, AJ;
Armant, M;
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(2017)
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
The New England Journal of Medicine
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10.1056/NEJMoa1700554.
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Ginn, SL;
Hallwirth, CV;
Liao, SHY;
Teber, ET;
Arthur, JW;
Wu, J;
Lee, HC;
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(2017)
Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.
Molecular Therapy - Nucleic Acids
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10.1016/j.omtn.2016.11.011.
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Hawkins, KE;
Moschidou, D;
Faccenda, D;
Wruck, W;
Martin-Trujillo, A;
Hau, KL;
Ranzoni, AM;
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(2017)
Human Amniocytes Are Receptive to Chemically Induced Reprogramming to Pluripotency.
Molecular Therapy
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10.1016/j.ymthe.2016.11.014.
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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
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10.1167/iovs.16-20608.
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Lee, PP;
Lobato-Márquez, D;
Pramanik, N;
Sirianni, A;
Daza-Cajigal, V;
Rivers, E;
Cavazza, A;
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(2017)
Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells.
Nature Communications
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Morris, EC;
Fox, T;
Chakraverty, R;
Tendeiro, R;
Snell, K;
Rivat, C;
Grace, S;
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(2017)
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
Blood
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10.1182/blood-2017-04-777136.
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Nowak, K;
Linzner, D;
Thrasher, AJ;
Lambert, PF;
Di, WL;
Burns, SO;
(2017)
Absence of γ-chain in keratinocytes alters chemokine secretion resulting in reduced immune cell recruitment.
Journal of Investigative Dermatology
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10.1016/j.jid.2017.05.024.
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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.
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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.
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Qasim, W;
Zhan, H;
Samarasinghe, S;
Adams, S;
Amrolia, P;
Stafford, S;
Butler, K;
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(2017)
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells.
Science Translational Medicine
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Rivers, E;
Thrasher, AJ;
(2017)
Wiskott-Aldrich syndrome protein: emerging mechanisms in immunity.
European Journal of Immunology
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10.1002/eji.201646715.
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Santilli, G;
Thrasher, AJ;
(2017)
A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).
Molecular Therapy
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10.1016/j.ymthe.2017.01.002.
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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.
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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.
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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.
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Standing, ASI;
Malinova, D;
Hong, Y;
Record, J;
Moulding, D;
Blundell, MP;
Nowak, K;
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(2017)
Autoinflammatory periodic fever, immunodeficiency, and thrombocytopenia (PFIT) caused by mutation in actinregulatory gene WDR1.
Journal of Experimental Medicine
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Thrasher, AJ;
(2017)
A Personal Reflection from London.
Human Gene Therapy
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Thrasher, AJ;
Williams, DA;
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Evolving Gene Therapy in Primary Immunodeficiency.
Molecular Therapy
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10.1016/j.ymthe.2017.03.018.
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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
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2016
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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
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10.1089/hum.2016.29033.ahb.
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Baptista, MA;
Keszei, M;
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Andersson, J;
Dahlberg, CI;
Worth, AJ;
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(2016)
Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.
Nature Communications
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Booth, C;
Gaspar, HB;
Thrasher, AJ;
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Treating Immunodeficiency through HSC Gene Therapy.
Trends In Molecular Medicine
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10.1016/j.molmed.2016.02.002.
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Chandra, A;
Zhang, F;
Gilmour, KC;
Webster, D;
Plagnol, V;
Kumararatne, DS;
Burns, SO;
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(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
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Angulo, I;
Plagnol, V;
Gilmour, KC;
Henriquez, F;
Curtis, J;
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(2016)
Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection.
Nature Communications
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Georgiadis, C;
(2016)
Development of Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa.
Doctoral thesis , UCL (University College London).
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Georgiadis, C;
Syed, F;
Petrova, A;
Abdul-Wahab, A;
Lwin, SM;
Farzaneh, F;
Chan, L;
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(2016)
Lentiviral Engineered Fibroblasts Expressing Codon Optimized COL7A1 Restore Anchoring Fibrils in RDEB.
Journal of Investigative Dermatology
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10.1038/jid.2015.364.
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Leon-Rico, D;
Aldea, M;
Sanchez-Baltasar, R;
Mesa-Nuñez, C;
Record, J;
Burns, SO;
Santilli, G;
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Construction of stable packaging cell lines for clinical lentiviral vector production.
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