Barry J. Byrne
Cited by
Cited by
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, 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
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 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
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
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
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
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
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
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
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 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
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
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
Acid a-glucosidase deficiency (glycogenosis type II, Pompe disease)
N Raben, P Plotz, BJ Byrne
Current molecular medicine 2 (2), 145-166, 2002
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