Format

Send to

Choose Destination
Nat Neurosci. 2019 Apr;22(4):524-528. doi: 10.1038/s41593-019-0352-0. Epub 2019 Mar 11.

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

Author information

1
Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
2
Department of Bioengineering, College of Engineering, Hanyang University, Seoul, Republic of Korea.
3
College of Pharmacy, Seoul National University, Seoul, Republic of Korea.
4
Department of Nanobiomedical Science, Dankook University, Cheonan, Republic of Korea.
5
Department of Biomedical Sciences, School of Veterinary Medicine and Institute for Regenerative Medicine, University of Pennsylvania, Philadelphia, PA, USA.
6
Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea. jpkim153@dongguk.edu.
7
Laboratory of Stem Cells & Cell Reprogramming, Department of Chemistry, Dongguk University, Seoul, Republic of Korea. jpkim153@dongguk.edu.

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.

PMID:
30858603
DOI:
10.1038/s41593-019-0352-0
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Nature Publishing Group
Loading ...
Support Center