Article
Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
Human molecular genetics - 1 Dec 2011
Ku Cristy A, Chiodo Vince A, Boye Sanford L, Goldberg Andrew F X, Li Tiansen, Hauswirth William W, Ramamurthy Visvanathan
Abstract excerpt
Defects in the photoreceptor-specific gene aryl hydrocarbon receptor interacting protein-like 1 (Aipl1) are associated with Leber congenital amaurosis (LCA), a childhood blinding disease with early-onset retinal degeneration and vision loss. Furthermore, Aipl1 defects are characterized at the most severe end of the LCA spectrum. The rapid photoreceptor degeneration and vision loss observed in the LCA patient...
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