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In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease
- Authors
- Park, Hanseul; Oh, Jungju; Shim, Gayong; Cho, Byounggook; Chang, Yujung; Kim, Siyoung; Baek, Soonbong; Kim, Hongwon; Shin, Jeain; Choi, Hwan; Yoo, Junsang; Kim, Junyeop; Jun, Won; Lee, Minhyung; Lengner, Christopher J.; Oh, Yu-Kyoung; Kim, Jongpil
- Issue Date
- Apr-2019
- Publisher
- NATURE PUBLISHING GROUP
- Citation
- NATURE NEUROSCIENCE, v.22, no.4, pp.524 - 528
- Indexed
- SCIE
SCOPUS
- Journal Title
- NATURE NEUROSCIENCE
- Volume
- 22
- Number
- 4
- Start Page
- 524
- End Page
- 528
- ISSN
- 1097-6256
- 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.
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Collections - 서울 공과대학 > 서울 생명공학과 > 1. Journal Articles
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Related Researcher
- Lee, Min hyung
- COLLEGE OF ENGINEERING (DEPARTMENT OF BIOENGINEERING)