| Literature DB >> 30858603 |
Hanseul Park1, Jungju Oh2, Gayong Shim3, Byounggook Cho1, Yujung Chang1, Siyoung Kim1, Soonbong Baek1, Hongwon Kim1, Jeain Shin1, Hwan Choi1, Junsang Yoo1, Junyeop Kim1, Won Jun4, Minhyung Lee2, Christopher J Lengner5, Yu-Kyoung Oh3, Jongpil Kim6,7.
Abstract
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.Entities:
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Year: 2019 PMID： 30858603 DOI： 10.1038/s41593-019-0352-0
Source DB: PubMed Journal: Nat Neurosci ISSN： 1097-6256 Impact factor: 24.884