Article
Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.
PLoS medicine - 1 Jun 2007
Aguirre Geoffrey K, Komáromy András M, Cideciyan Artur V, Brainard David H, Aleman Tomas S, Roman Alejandro J, Avants Brian B, Gee James C, Korczykowski Marc, Hauswirth William W, Acland Gregory M, Aguirre Gustavo D, Jacobson Samuel G
Abstract excerpt
BACKGROUND: RPE65 is an essential molecule in the retinoid-visual cycle, and RPE65 gene mutations cause the congenital human blindness known as Leber congenital amaurosis (LCA). Somatic gene therapy delivered to the retina of blind dogs with an RPE65 mutation dramatically restores retinal physiology and has sparked international interest in human treatment trials for this incurable disease. An unanswered question...
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