Article
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2011
Dalkara Deniz, Kolstad Kathleen D, Guerin Karen I, Hoffmann Natalie V, Visel Meike, Klimczak Ryan R, Schaffer David V, Flannery John G
Abstract excerpt
Mutations in over 80 identified genes can induce apoptosis in photoreceptors, resulting in blindness with a prevalence of 1 in 3,000 individuals. This broad genetic heterogeneity of disease impacting a wide range of photoreceptor functions renders the design of gene-specific therapies for photoreceptor degeneration impractical and necessitates the development of mutation-independent treatments to slow...
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