Article
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 - 5 Feb 2013
Cideciyan Artur V, Jacobson Samuel G, Beltran William A, Sumaroka Alexander, Swider Malgorzata, Iwabe Simone, Roman Alejandro J, Olivares Melani B, Schwartz Sharon B, Komáromy András M, Hauswirth William W, Aguirre Gustavo D
Abstract excerpt
Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both dysfunction and degeneration of photoreceptors. Clinical trials with gene augmentation therapy have shown partial reversal of the dysfunction, but the effects on the degeneration are not known. We evaluated the consequences of gene therapy on...
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