| First Author | Park H | Year | 2019 |
| Journal | Nat Neurosci | Volume | 22 |
| Issue | 4 | Pages | 524-528 |
| PubMed ID | 30858603 | Mgi Jnum | J:277515 |
| Mgi Id | MGI:6313564 | Doi | 10.1038/s41593-019-0352-0 |
| Citation | Park H, et al. (2019) In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease. Nat Neurosci 22(4):524-528 |
| abstractText | 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 (Abeta)-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. |
