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| Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield S Zolotukhin, BJ Byrne, E Mason, I Zolotukhin, M Potter, K Chesnut, ... Gene therapy 6 (6), 973-985, 1999 | 1605 | 1999 |
| Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial WW Hauswirth, TS Aleman, S Kaushal, AV Cideciyan, SB Schwartz, ... Human gene therapy 19 (10), 979-990, 2008 | 1206 | 2008 |
| Recruitment of human muscleblind proteins to (CUG) n expansions associated with myotonic dystrophy JW Miller, CR Urbinati, P Teng‐umnuay, MG Stenberg, BJ Byrne, ... The EMBO journal, 2000 | 1108 | 2000 |
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| Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease PS Kishnani, D Corzo, M Nicolino, B Byrne, H Mandel, WL Hwu, N Leslie, ... Neurology 68 (2), 99-109, 2007 | 913 | 2007 |
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics AV Cideciyan, TS Aleman, SL Boye, SB Schwartz, S Kaushal, AJ Roman, ... Proceedings of the National Academy of Sciences 105 (39), 15112-15117, 2008 | 828 | 2008 |
| Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein PD Kessler, GM Podsakoff, X Chen, SA McQuiston, PC Colosi, LA Matelis, ... Proceedings of the National Academy of Sciences 93 (24), 14082-14087, 1996 | 799 | 1996 |
| Pompe disease diagnosis and management guideline PS Kishnani, RD Steiner, D Bali, K Berger, BJ Byrne, LE Case, ... Genetics in Medicine 8 (5), 267-288, 2006 | 791 | 2006 |
| Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years SG Jacobson, AV Cideciyan, R Ratnakaram, E Heon, SB Schwartz, ... Archives of ophthalmology 130 (1), 9-24, 2012 | 787 | 2012 |
| Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors S Zolotukhin, M Potter, I Zolotukhin, Y Sakai, S Loiler, TJ Fraites Jr, ... Methods 28 (2), 158-167, 2002 | 676 | 2002 |
| Current clinical applications of in vivo gene therapy with AAVs JR Mendell, SA Al-Zaidy, LR Rodino-Klapac, K Goodspeed, SJ Gray, ... Molecular Therapy 29 (2), 464-488, 2021 | 587 | 2021 |
| Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy JR Mendell, N Goemans, LP Lowes, LN Alfano, K Berry, J Shao, EM Kaye, ... Annals of neurology 79 (2), 257-271, 2016 | 536 | 2016 |
| Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo CA Pacak, CS Mah, BD Thattaliyath, TJ Conlon, MA Lewis, DE Cloutier, ... Circulation research 99 (4), e3-e9, 2006 | 488 | 2006 |
| Effect of intracoronary delivery of autologous bone marrow mononuclear cells 2 to 3 weeks following acute myocardial infarction on left ventricular function: the LateTIME … JH Traverse, TD Henry, SG Ellis, CJ Pepine, JT Willerson, DXM Zhao, ... Jama 306 (19), 2110-2119, 2011 | 475 | 2011 |
| Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year AV Cideciyan, WW Hauswirth, TS Aleman, S Kaushal, SB Schwartz, ... Human gene therapy 20 (9), 999-1004, 2009 | 428 | 2009 |
| Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy ML Brantly, JD Chulay, L Wang, C Mueller, M Humphries, LT Spencer, ... Proceedings of the National Academy of Sciences 106 (38), 16363-16368, 2009 | 390 | 2009 |
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors S Song, M Morgan, T Ellis, A Poirier, K Chesnut, J Wang, M Brantly, ... Proceedings of the National Academy of Sciences 95 (24), 14384-14388, 1998 | 362 | 1998 |
| Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease M Nicolino, B Byrne, JE Wraith, N Leslie, H Mandel, DR Freyer, GL Arnold, ... Genetics in medicine 11 (3), 210-219, 2009 | 357 | 2009 |
| Acid a-glucosidase deficiency (glycogenosis type II, Pompe disease) N Raben, P Plotz, BJ Byrne Current molecular medicine 2 (2), 145-166, 2002 | 332 | 2002 |
David D. FullerUniversity of FloridaVerified email at phhp.ufl.edu
