Article

Allele-specific gene editing approach for vision loss restoration in <i>RHO</i> -associated Retinitis Pigmentosa

2022-11-16

Abstract excerpt

Mutant RHO is the most frequent genetic cause of autosomal dominant retinitis pigmentosa. Here, we developed an allele-specific gene editing therapeutic drug to selectively target the human T17M RHO mutant allele while leaving the wild-type RHO allele intact for the first time. We identified a Staphylococcus aureus Cas9 (SaCas9) guide RNA that was highly active and specific to the human T17M RHO allele. In v...

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Literature Corpus work: 7c89a591-438a-5c60-b873-70b68ea0770e
DOI: 10.1101/2022.11.16.516784

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