LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile
Rajeshwari D. Koilkonda(University of Miami), Josephine M. Guy(University of Miami), Sanford L. Boye(University of Florida), Thomas J. Conlon(Adelson Foundation), Lauren Renner(Oregon National Primate Research Center), Vince A. Chiodo, Kirsten Erger(University of Florida), Alfred S. Lewin(University of Florida), Martha Neuringer(Oregon National Primate Research Center), Vittorio Porciatti(University of Miami), William W. Hauswirth(University of Florida), Carol J. Detrisac(Charles River Associates), Hong Yu(University of Miami), Venu Talla(University of Miami), William J. Feuer(University of Miami)
Cited by 67
Related Papers
Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
|Human Gene Therapy|2008|950
Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo
|Circulation Research|2006|400
Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism
|Journal of Virology|2000|392
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
|Proceedings of the National Academy of Sciences|2009|322
Treatment Outcomes in the Primary Tube Versus Trabeculectomy Study after 1 Year of Follow-up
|Ophthalmology|2018|320