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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 | 1082 | 2000 |
Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization MB Grant, WS May, S Caballero, GAJ Brown, SM Guthrie, RN Mames, ... Nature medicine 8 (6), 607-612, 2002 | 933 | 2002 |
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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 | 813 | 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 | 794 | 1996 |
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 | 776 | 2012 |
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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 | 514 | 2016 |
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 | 503 | 2021 |
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 | 477 | 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 | 467 | 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 | 423 | 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 | 379 | 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 | 357 | 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 | 343 | 2009 |
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