Melani Olivares

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

Archives of Ophthalmology · 2012 · 529 citations

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Proceedings of the National Academy of Sciences of the United States of America · 2013 · 369 citations