Gene therapy comes of ageCynthia E. Dunbar, Michel Sadelain, Katherine A. High et al.|Science|2018Cited by 1.4k
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialAlbert M. Maguire, Jean Bennett, Ann Fulton et al.|The Lancet|2009Cited by 857
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector AdministrationFrancesca Simonelli, Alberto Auricchio, Albert M. Maguire et al.|Molecular Therapy|2009Cited by 551
AAV2 Gene Therapy Readministration in Three Adults with Congenital BlindnessJean Bennett, Albert M. Maguire, Manzar Ashtari et al.|Science Translational Medicine|2012Cited by 390
Intramuscular Administration of AAV1-Lipoprotein Lipase <sup>S447X</sup> Lowers Triglycerides in Lipoprotein Lipase–Deficient PatientsErik S.G. Stroes, Jan Albert Kuivenhoven, Melchior C. Nierman et al.|Arteriosclerosis Thrombosis and Vascular Biology|2008Cited by 208