Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration

Undesired chromosomal translocations, vector integrations, and large deletions remain a problem for therapeutic gene editing in vivo. Here, the authors compare the CRISPR-Cas9TX variant with CRISPR-Cas9 and show elimination of chromosomal translocations and reduction of AVV integration when targetin...

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Bibliographic Details
Main Authors: Jianhang Yin, Kailun Fang, Yanxia Gao, Liqiong Ou, Shaopeng Yuan, Changchang Xin, Weiwei Wu, Wei-wei Wu, Jiaxu Hong, Hui Yang, Jiazhi Hu
Format: Article
Language:English
Published: Nature Portfolio 2022-12-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-35640-4