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

V. Cideciyan, Artur; G. Jacobson, Samuel; A. Beltran, William; Sumaroka, Alexander; Świder, Małgorzata; Iwabe, Simone; J. Román, Alejandro; B. Olivares, Melani; Schwartz, Sharon; M. Komáromy, András; W. Hauswirth, William; D. Aguirre, Gustavo

doi:10.1073/pnas.1218933110

Public

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

Published in Proceedings of the National Academy of Sciences • Jan 22, 2013

Authors:

Artur V. Cideciyan

Samuel G. Jacobson

William A. Beltran

Abstract

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...

Subject

RPE65

Retinal degeneration

Visual phototransduction

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