In vivo precision base editing to rescue mouse models of disease

In vivo precision base editing to rescue mouse models of disease

CRISPR base editing enables precise, irreversible base conversions without inducing double-stranded breaks (DSBs) and has gained significant attention in recent years. By converting cytosine to thymine (C→T) or adenine to guanine (A→G), base editors (BEs) efficiently correct pathogenic single-nucleo...

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Bibliographic Details
Main Authors: Aaron Schindeler, Julian Chu, Christal Au-Yeung, Hsien-Yin Kao, Samantha L. Ginn, Alexandra K. O’Donohue
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA editing
gene therapy
gene editing
CRISPR-Cas9
base editing
viral vectors
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001763
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