Article
Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration.
Investigative ophthalmology & visual science - 1 Oct 2015
Murray Susan F, Jazayeri Ali, Matthes Michael T, Yasumura Douglas, Yang Haidong, Peralta Raechel, Watt Andy, Freier Sue, Hung Gene, Adamson Peter S, Guo Shuling, Monia Brett P, LaVail Matthew M, McCaleb Michael L
Abstract excerpt
PURPOSE: To preserve photoreceptor cell structure and function in a rodent model of retinitis pigmentosa with P23H rhodopsin by selective inhibition of the mutant rhodopsin allele using a second generation antisense oligonucleotide (ASO). METHODS: Wild-type mice and rats were treated with ASO by intravitreal (IVT) injection and rhodopsin mRNA and protein expression were measured. Transgenic rats expressing the...
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