Article
Whole-brain in vivo base editing reverses behavioral changes in Mef2c-mutant mice.
Nature neuroscience - 1 Jan 2024
Li Wei-Ke, Zhang Shu-Qian, Peng Wan-Ling, Shi Yu-Han, Yuan Bo, Yuan Yi-Ting, Xue Zhen-Yu, Wang Jin-Cheng, Han Wen-Jian, Chen Zhi-Fang, Shan Shi-Fang, Xue Bi-Qing, Chen Jin-Long, Zhang Cheng, Zhu Shu-Jia, Tai Yi-Lin, Cheng Tian-Lin, Qiu Zi-Long
Abstract excerpt
Whole-brain genome editing to correct single-base mutations and reduce or reverse behavioral changes in animal models of autism spectrum disorder (ASD) has not yet been achieved. We developed an apolipoprotein B messenger RNA-editing enzyme, catalytic polypeptide-embedded cytosine base editor (AeCBE) system for converting C·G to T·A base pairs. We demonstrate its effectiveness by targeting AeCBE to an...
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