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Article

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin.

Genes - 26 Jul 2022

Kaukonen Maria, McClements Michelle E, MacLaren Robert E

Abstract excerpt

Retinitis pigmentosa (RP) is the most common group of inherited retinal degenerations and pathogenic variants in the Rhodopsin (RHO) gene are major cause for autosomal dominant RP (adRP). Despite extensive attempts to treat RHO-associated adRP, standardized curative treatment is still lacking. Recently developed base editors offer an exciting opportunity to correct pathogenic single nucleotide variants and are...

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Identifiers

PMID: 35893064
DOI: 10.3390/genes13081327
PMCID: PMC9330713

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