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

Autor: Jianhang Yin, Kailun Fang, Yanxia Gao, Liqiong Ou, Shaopeng Yuan, Changchang Xin, Weiwei Wu, Wei-wei Wu, Jiaxu Hong, Hui Yang, Jiazhi Hu
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Nature Communications, Vol 13, Iss 1, Pp 1-8 (2022)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/s41467-022-35640-4
Popis: 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 targeting Vegfa for the treatment of age-related macular degeneration in a mouse model.
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