Chimeric antigen receptor T cells for sustained remissions in leukemia SL Maude, N Frey, PA Shaw, R Aplenc, DM Barrett, NJ Bunin, A Chew, ... New England Journal of Medicine 371 (16), 1507-1517, 2014 | 6034 | 2014 |
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 | 4221 | 2013 |
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 | 2630 | 2008 |
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 | 2466 | 2006 |
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 | 1766 | 2017 |
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 | 1077 | 2009 |
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 | 759 | 2017 |
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 | 730 | 2010 |
Chimeric antigen receptor T cells against CD19 for multiple myeloma AL Garfall, MV Maus, WT Hwang, SF Lacey, YD Mahnke, JJ Melenhorst, ... New England Journal of Medicine 373 (11), 1040-1047, 2015 | 675 | 2015 |
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 | 513 | 2016 |
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 | 505 | 2009 |
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 | 479 | 2012 |
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 | 409 | 2013 |
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 | 364 | 2013 |
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 | 353 | 2010 |
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 | 342 | 2019 |
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 | 323 | 2008 |
Progress and challenges in viral vector manufacturing JCM Van Der Loo, JF Wright Human molecular genetics 25 (R1), R42-R52, 2016 | 297 | 2016 |
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 | 279 | 2007 |
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 | 277 | 2011 |