清脆的
神经科学
基因组编辑
体内
生物
疾病
Cas9
阿尔茨海默病
基因
遗传增强
医学
遗传学
病理
作者
Hanseul Park,Jungju Oh,Gayong Shim,Byounggook Cho,Yu‐Jung Chang,Siyoung Kim,Soonbong Baek,Hongwon Kim,Jeain Shin,Hwan Geun Choi,Jang-Yong Yoo,Junyeop Kim,Won Jun,Minhyung Lee,Christopher J. Lengner,Yu‐Kyoung Oh,Jongpil Kim
标识
DOI:10.1038/s41593-019-0352-0
摘要
In vivo gene editing in post-mitotic neurons of the adult brain may be a useful strategy for treating neurological diseases. Here, we develop CRISPR-Cas9 nanocomplexes and show they were effective in the adult mouse brain, with minimal off-target effects. Using this system to target Bace1 suppressed amyloid beta (Aβ)-associated pathologies and cognitive deficits in two mouse models of Alzheimer's disease. These results broaden the potential application of CRISPR-Cas9 systems to neurodegenerative diseases.
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