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Paper

In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease
Author
Hanseul Park, Jongju Oh, Gayong Shim, Byounggook Cho, Yujung Chang, Siyoung Kim, Soonbong Baek, HongWon Kim, Jeain Shin , Hwan Choi, JunSang Yoo, Junyeop Kim, Won Jun, Minhyung Lee, Christopher Lengner, Yu-Kyoung Oh, Jongpil Kim
Journal
Nature Neuroscience (SCIE)
Vol
22 (4)
Page
524-528
Year
2019
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.