Preclinical Potency and Safety Studies of an AAV2-Mediated Gene Therapy Vector for the Treatment of <i>MERTK</i> Associated Retinitis PigmentosaThomas J. Conlon, William W. Hauswirth, Ji‐Jing Pang et al.|Human Gene Therapy Clinical Development|2013Cited by 95
Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-GlucosidaseThomas J. Conlon, Barry J. Byrne, Kirsten Erger et al.|Human Gene Therapy Clinical Development|2013Cited by 21