Article
RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model.
PloS one - 1 Jan 2013
Jiang Li, Li Tansy Z, Boye Shannon E, Hauswirth William W, Frederick Jeanne M, Baehr Wolfgang
Abstract excerpt
Dominant mutations occurring in the high-affinity Ca(2+)-binding sites (EF-hands) of the GUCA1A gene encoding guanylate cyclase-activating protein 1 (GCAP1) cause slowly progressing cone-rod dystrophy (CORD) in a dozen families worldwide. We developed a nonallele-specific adeno-associated virus (AAV)-based RNAi knockdown strategy to rescue the retina degeneration caused by GCAP1 mutations. We generated three...
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