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, Issam McDoom et al.|Human Gene Therapy Clinical Development|2013Cited by 95
Long-term Preservation of Cone Photoreceptors and Restoration of Cone Function by Gene Therapy in the Guanylate Cyclase-1 Knockout (GC1KO) MouseSanford L. Boye, Kirsten Erger, Renee C. Ryals et al.|Investigative Ophthalmology & Visual Science|2011Cited by 63
Efficient Hepatic Delivery and Expression from a Recombinant Adeno-associated Virus 8 Pseudotyped α1-Antitrypsin VectorThomas J. Conlon, Terence R. Flotte, Travis Cossette et al.|Molecular Therapy|2005Cited by 56
Stability and Safety of an AAV Vector for Treating <i>RPGR-ORF15</i> X-Linked Retinitis PigmentosaWen‐Tao Deng, William W. Hauswirth, Frank M. Dyka et al.|Human Gene Therapy|2015Cited by 55
Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene TherapyAllison M. Keeler, Terence R. Flotte, Thomas J. Conlon et al.|Molecular Therapy|2012Cited by 25