@article{4e43d451886745cc856c1bc373fe0eee,
title = "In vivo neuronal gene editing via CRISPR–Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer{\textquoteright}s disease",
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{\textquoteright}s disease. These results broaden the potential application of CRISPR–Cas9 systems to neurodegenerative diseases.",
author = "Hanseul Park and Jungju Oh and Gayong Shim and Byounggook Cho and Yujung Chang and Siyoung Kim and Soonbong Baek and Hongwon Kim and Jeain Shin and Hwan Choi and Junsang Yoo and Junyeop Kim and Won Jun and Minhyung Lee and Lengner, {Christopher J.} and Oh, {Yu Kyoung} and Jongpil Kim",
note = "Publisher Copyright: {\textcopyright} 2019, The Author(s), under exclusive licence to Springer Nature America, Inc.",
year = "2019",
month = apr,
day = "1",
doi = "10.1038/s41593-019-0352-0",
language = "English",
volume = "22",
pages = "524--528",
journal = "Nature Neuroscience",
issn = "1097-6256",
publisher = "Nature Research",
number = "4",
}