Article

Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseases

2025-08-06

Abstract excerpt

<h4>ABSTRACT</h4> Site-directed RNA editing, especially RNA base editing, allows for specific manipulation of RNA sequences, making it a useful approach for the correction of pathogenic mutations. Correction of RNA transcripts allows therapeutic gene editing in a safe and reversible manner and avoids permanent alterations in the genome. RNA-targeting CRISPR-Cas nucleases (e.g., CRISPR-Cas13) enable delivery withi...

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Literature Corpus work: fedbeb62-3ea5-5633-bfb4-d6fce814f55f
DOI: 10.1101/2025.08.06.668808

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