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Efficient prime editing in mouse brain, liver and heart with dual AAVs

4 May 2023

Abstract excerpt

Realizing the promise of prime editing for the study and treatment of genetic disorders requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the identification of bottlenecks limiting adeno-associated virus (AAV)-mediated prime editing in vivo and the development of AAV-PE vectors with increased PE expression, prime editing guide RNA stability and modulation of DNA repair. The...

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PMID: 37142705
DOI: 10.1038/s41587-023-01758-z

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