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

Title
In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease
Author
이민형
Keywords
MICE; EXPRESSION; PROTEIN; MUSCLE
Issue Date
2019-04
Publisher
NATURE PUBLISHING GROUP
Citation
NATURE NEUROSCIENCE, v. 22, NO 4, Page. 524-528
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 Bacel suppressed amyloid beta (A beta)-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.
URI
https://www.nature.com/articles/s41593-019-0352-0http://repository.hanyang.ac.kr/handle/20.500.11754/109955
ISSN
1097-6256; 1546-1726
DOI
10.1038/s41593-019-0352-0
Appears in Collections:
COLLEGE OF ENGINEERING[S](공과대학) > BIOENGINEERING(생명공학과) > Articles
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