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Article

CRISPRi-Mediated Treatment of Dominant Rhodopsin-Associated Retinitis Pigmentosa.

The CRISPR journal - 1 Dec 2023

Burnight Erin R, Wiley Luke A, Mullin Nathaniel K, Adur Malavika K, Lang Mallory J, Cranston Cathryn M, Jiao Chunhua, Russell Stephen R, Sohn Elliot H, Han Ian C, Ross Jason W, Stone Edwin M, Mullins Robert F, Tucker Budd A

Abstract excerpt

Rhodopsin (RHO) mutations such as Pro23His are the leading cause of dominantly inherited retinitis pigmentosa in North America. As with other dominant retinal dystrophies, these mutations lead to production of a toxic protein product, and treatment will require knockdown of the mutant allele. The purpose of this study was to develop a CRISPR-Cas9-mediated transcriptional repression strategy using catalytically...

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Identifiers

PMID: 38108516
DOI: 10.1089/crispr.2023.0039
PMCID: PMC11304754

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