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


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2019-04

AUTHORS

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

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. More... »

PAGES

524-528

References to SciGraph publications

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/s41593-019-0352-0

DOI

http://dx.doi.org/10.1038/s41593-019-0352-0

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1112678127

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/30858603


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