Gustavo D. Aguirreb

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

Artur V. Cideciyana, Samuel G. Jacobsona, +9 authors Gustavo D. Aguirreb
Proceedings of the National Academy of Sciences…
2013

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… (More)

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