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J Fraser Wright
J Fraser Wright
Adresse e-mail validée de stanford.edu
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Chimeric antigen receptor–modified T cells for acute lymphoid leukemia
SA Grupp, M Kalos, D Barrett, R Aplenc, DL Porter, SR Rheingold, ...
New England Journal of Medicine 368 (16), 1509-1518, 2013
40082013
Safety and efficacy of gene transfer for Leber's congenital amaurosis
AM Maguire, F Simonelli, EA Pierce, EN Pugh Jr, F Mingozzi, J Bennicelli, ...
New England Journal of Medicine 358 (21), 2240-2248, 2008
25572008
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
CS Manno, GF Pierce, VR Arruda, B Glader, M Ragni, JJE Rasko, ...
Nature medicine 12 (3), 342-347, 2006
23552006
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
S Russell, J Bennett, JA Wellman, DC Chung, ZF Yu, A Tillman, J Wittes, ...
The Lancet 390 (10097), 849-860, 2017
15772017
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
AM Maguire, KA High, A Auricchio, JF Wright, EA Pierce, F Testa, ...
The Lancet 374 (9701), 1597-1605, 2009
10272009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
F Simonelli, AM Maguire, F Testa, EA Pierce, F Mingozzi, JL Bennicelli, ...
Molecular Therapy 18 (3), 643-650, 2010
7142010
Hemophilia B gene therapy with a high-specific-activity factor IX variant
LA George, SK Sullivan, A Giermasz, JEJ Rasko, BJ Samelson-Jones, ...
New England Journal of Medicine 377 (23), 2215-2227, 2017
6942017
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
CW Christine, PA Starr, PS Larson, JL Eberling, WJ Jagust, RA Hawkins, ...
Neurology 73 (20), 1662-1669, 2009
4792009
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase …
J Bennett, J Wellman, KA Marshall, S McCague, M Ashtari, ...
The Lancet 388 (10045), 661-672, 2016
4752016
AAV2 gene therapy readministration in three adults with congenital blindness
J Bennett, M Ashtari, J Wellman, KA Marshall, LL Cyckowski, DC Chung, ...
Science translational medicine 4 (120), 120ra15-120ra15, 2012
4652012
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2
F Testa, AM Maguire, S Rossi, EA Pierce, P Melillo, K Marshall, S Banfi, ...
Ophthalmology 120 (6), 1283-1291, 2013
3982013
Overcoming preexisting humoral immunity to AAV using capsid decoys
F Mingozzi, XM Anguela, G Pavani, Y Chen, RJ Davidson, DJ Hui, ...
Science translational medicine 5 (194), 194ra92-194ra92, 2013
3372013
High AAV vector purity results in serotype-and tissue-independent enhancement of transduction efficiency
E Ayuso, F Mingozzi, J Montane, X Leon, XM Anguela, V Haurigot, ...
Gene therapy 17 (4), 503-510, 2010
3352010
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
J Bennicelli, JF Wright, A Komaromy, JB Jacobs, B Hauck, O Zelenaia, ...
Molecular Therapy 16 (3), 458-465, 2008
3022008
Efficacy, safety, and durability of voretigene neparvovec-rzyl in RPE65 mutation–associated inherited retinal dystrophy: results of phase 1 and 3 trials
AM Maguire, S Russell, JA Wellman, DC Chung, ZF Yu, A Tillman, ...
Ophthalmology 126 (9), 1273-1285, 2019
2932019
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
F Mingozzi, NC Hasbrouck, E Basner-Tschakarjan, SA Edmonson, DJ Hui, ...
Blood, The Journal of the American Society of Hematology 110 (7), 2334-2341, 2007
2682007
Progress and challenges in viral vector manufacturing
JCM Van Der Loo, JF Wright
Human molecular genetics 25 (R1), R42-R52, 2016
2592016
Assessing the potential for AAV vector genotoxicity in a murine model
H Li, N Malani, SR Hamilton, A Schlachterman, G Bussadori, ...
Blood, The Journal of the American Society of Hematology 117 (12), 3311-3319, 2011
2552011
Quantification of adeno-associated virus particles and empty capsids by optical density measurement
PH Smith, S Parthasarathy, J Isaacs, S Vijay, J Kieran, SK Powell, ...
Molecular Therapy 7 (1), 122-128, 2003
2242003
Manufacturing and characterizing AAV-based vectors for use in clinical studies
JF Wright
Gene therapy 15 (11), 840-848, 2008
2232008
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