Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
Jared Silver(University of Florida), Terence R. Flotte(University of Massachusetts Chan Medical School), Melissa E. Elder, Pedro E. Cruz, Amy Wright(University of Florida), Arun Srivastava(University of Florida), Thomas J. Conlon(Adelson Foundation)
Cited by 7
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
Formation of a Human β-Cell Population within Pancreatic Islets Is Set Early in Life
|The Journal of Clinical Endocrinology & Metabolism|2012|340
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