AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins


Gene therapy for inherited retinal degeneration in which expression of a mutant allele has a gain-of-function effect on photoreceptor cells is likely to depend on efficient silencing of the mutated allele. Peripherin-2 (Prph2, also known as peripherin/RDS) is an abundantly expressed photoreceptor-specific gene. In humans, gain-of-function mutations in PRPH2… (More)
DOI: 10.1038/gt.2009.162


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