|Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart|
C Toma, MF Pittenger, KS Cahill, BJ Byrne, PD Kessler
Circulation 105 (1), 93-98, 2002
|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
|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
|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 19 (17), 4439-4448, 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
|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
|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
|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
|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
|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
|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
|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
|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
|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
|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
|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
|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
|In vivo murine left ventricular pressure-volume relations by miniaturized conductance micromanometry|
D Georgakopoulos, WA Mitzner, CH Chen, BJ Byrne, HD Millar, JM Hare, ...
American Journal of Physiology-Heart and Circulatory Physiology 274 (4 …, 1998
|Acid a-glucosidase deficiency (glycogenosis type II, Pompe disease)|
N Raben, P Plotz, BJ Byrne
Current molecular medicine 2 (2), 145-166, 2002
|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