Pompe disease gene therapy
Barry J. Byrne(University of Florida), Cathryn Mah(Anna Needs Neuroblastoma Answers), Shelley Collins(University of Florida), Thomas J. Conlon(Adelson Foundation), Sushrusha Nayak, Darin J. Falk, David D. Fuller(Brenau University), Melissa E. Elder, Brian Cleaver(University of Florida), Barbara K. Smith(University of Florida), Stacy Porvasnik(University of Florida), Christina A. Pacak(University of Florida), Roland W. Herzog(Heidelberg University), Mai K. ElMallah(Duke University), Anatole D. Martin(University of Florida Health), Saleem Islam(University of Florida), Lee Ann Lawson(University of Florida), Denise A Cloutier(University of Florida), Nathalie Clément(University of Florida)
Cited by 93
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