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Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration
Author(s) -
Susan Murray,
Ali Jazayeri,
Michael T. Matthes,
Douglas Yasumura,
Haidong Yang,
Raechel Peralta,
Andy Watt,
Sue Freier,
Gene Hung,
Peter Adamson,
Shuling Guo,
Brett P. Monia,
Matthew M. LaVail,
Michael L. McCaleb
Publication year - 2015
Publication title -
investigative ophthalmology and visual science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.935
H-Index - 218
eISSN - 1552-5783
pISSN - 0146-0404
DOI - 10.1167/iovs.15-16400
Subject(s) - rhodopsin , retinitis pigmentosa , electroretinography , microbiology and biotechnology , outer nuclear layer , retinal degeneration , transgene , photoreceptor cell , biology , erg , chemistry , retinal , gene , biochemistry
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).

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