Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialAlbert M. Maguire, Jean Bennett, Eric A. Pierce 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