In vivo neuronal gene editing via CRISPR–Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer’s disease

Hanseul Park, Jungju 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 J. Lengner, Yu Kyoung Oh, Jongpil Kim

Research output: Contribution to journalArticlepeer-review

208 Scopus citations

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.

Original languageEnglish
Pages (from-to)524-528
Number of pages5
JournalNature Neuroscience
Volume22
Issue number4
DOIs
StatePublished - 1 Apr 2019

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