Article

Treatment of a genetic liver disease in mice through transient prime editor expression

2024-01-24

Abstract excerpt

Prime editing is a versatile genome editing technology that does not rely on DNA double-strand break formation and homology-directed repair (HDR). This makes it a promising tool for correcting pathogenic mutations in tissues consisting predominantly of postmitotic cells, such as the liver. While recent studies have already demonstrated proof-of-concept for in vivo prime editing, the use of viral delivery vectors...

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Literature Corpus work: 5c533819-69a6-5f72-bfef-c82b5b72780a
DOI: 10.1101/2024.01.22.575834

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