Παρακολούθηση
adrian thrasher
adrian thrasher
UCL/GOSH
Η διεύθυνση ηλεκτρονικού ταχυδρομείου έχει επαληθευτεί στον τομέα ucl.ac.uk
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Effect of gene therapy on visual function in Leber's congenital amaurosis
JWB Bainbridge, AJ Smith, SS Barker, S Robbie, R Henderson, ...
New England Journal of Medicine 358 (21), 2231-2239, 2008
24682008
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
SJ Howe, MR Mansour, K Schwarzwaelder, C Bartholomae, M Hubank, ...
The Journal of clinical investigation 118 (9), 2008
15112008
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
S Hacein-Bey-Abina, F Le Deist, F Carlier, C Bouneaud, C Hue, ...
New england journal of medicine 346 (16), 1185-1193, 2002
14862002
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
MG Ott, M Schmidt, K Schwarzwaelder, S Stein, U Siler, U Koehl, H Glimm, ...
Nature medicine 12 (4), 401-409, 2006
14202006
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
W Qasim, H Zhan, S Samarasinghe, S Adams, P Amrolia, S Stafford, ...
Science translational medicine 9 (374), eaaj2013, 2017
10202017
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
S Stein, MG Ott, S Schultze-Strasser, A Jauch, B Burwinkel, A Kinner, ...
Nature medicine 16 (2), 198-204, 2010
9382010
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
HB Gaspar, KL Parsley, S Howe, D King, KC Gilmour, J Sinclair, G Brouns, ...
The Lancet 364 (9452), 2181-2187, 2004
8572004
A robust model for read count data in exome sequencing experiments and implications for copy number variant calling
V Plagnol, J Curtis, M Epstein, KY Mok, E Stebbings, S Grigoriadou, ...
Bioinformatics 28 (21), 2747-2754, 2012
7572012
The cytoplasm of living cells behaves as a poroelastic material
E Moeendarbary, L Valon, M Fritzsche, AR Harris, DA Moulding, ...
Nature materials 12 (3), 253-261, 2013
7282013
High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen …
C Demaison, K Parsley, G Brouns, M Scherr, K Battmer, C Kinnon, M Grez, ...
Human gene therapy 13 (7), 803-813, 2002
6352002
Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
F Eichler, C Duncan, PL Musolino, PJ Orchard, S De Oliveira, AJ Thrasher, ...
New England Journal of Medicine 377 (17), 1630-1638, 2017
6062017
Effective gene therapy with nonintegrating lentiviral vectors
RJ Yánez-Munoz, KS Balaggan, A MacNeil, SJ Howe, M Schmidt, ...
Nature medicine 12 (3), 348-353, 2006
5852006
The European Society for Immunodeficiencies (ESID) registry working definitions for the clinical diagnosis of inborn errors of immunity
MG Seidel, G Kindle, B Gathmann, I Quinti, M Buckland, J van Montfrans, ...
The Journal of Allergy and Clinical Immunology: In Practice 7 (6), 1763-1770, 2019
5552019
The wiskott-aldrich syndrome
HD Ochs, AJ Thrasher
Journal of Allergy and Clinical Immunology 117 (4), 725-738, 2006
5182006
Clinical features and outcome of patients with IRAK-4 and MyD88 deficiency
C Picard, H Von Bernuth, P Ghandil, M Chrabieh, O Levy, PD Arkwright, ...
Medicine 89 (6), 403-425, 2010
5112010
Whole-genome sequencing of patients with rare diseases in a national health system
E Turro, WJ Astle, K Megy, S Gräf, D Greene, O Shamardina, HL Allen, ...
Nature 583 (7814), 96-102, 2020
5032020
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors
U Modlich, S Navarro, D Zychlinski, T Maetzig, S Knoess, MH Brugman, ...
Molecular Therapy 17 (11), 1919-1928, 2009
4892009
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency
S Hacein-Bey-Abina, SY Pai, HB Gaspar, M Armant, CC Berry, S Blanche, ...
New England Journal of Medicine 371 (15), 1407-1417, 2014
4822014
WASP: a key immunological multitasker
AJ Thrasher, SO Burns
Nature Reviews Immunology 10 (3), 182-192, 2010
4682010
Comprehensive rare variant analysis via whole-genome sequencing to determine the molecular pathology of inherited retinal disease
KJ Carss, G Arno, M Erwood, J Stephens, A Sanchis-Juan, S Hull, K Megy, ...
The American Journal of Human Genetics 100 (1), 75-90, 2017
4672017
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