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 TrialWilliam W. Hauswirth, Samuel G. Jacobson, Tomás S. Alemán et al.|Human Gene Therapy|2008Cited by 950
Human <i>RPE65</i> Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 YearArtur V. Cideciyan, Samuel G. Jacobson, Thomas J. Conlon et al.|Human Gene Therapy|2009Cited by 312
Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal InjectionSamuel G. Jacobson, William W. Hauswirth, Gregory M. Acland et al.|Molecular Therapy|2006Cited by 223
Vision 1 Year after Gene Therapy for Leber's Congenital AmaurosisArtur V. Cideciyan, Samuel G. Jacobson, William W. Hauswirth et al.|New England Journal of Medicine|2009Cited by 199
Safety in Nonhuman Primates of Ocular AAV2- <i>RPE65</i> , a Candidate Treatment for Blindness in Leber Congenital AmaurosisSamuel G. Jacobson, William W. Hauswirth, Sanford L. Boye et al.|Human Gene Therapy|2006Cited by 147