Safeguarding genome integrity during gene-editing therapy in a mouse model of age-related macular degeneration
oleh: 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 |
|---|---|
| Diterbitkan: | Nature Portfolio 2022-12-01 |
Deskripsi
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.