A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.
Document Type
Journal Article
Publication Date
3-1-2016
Journal
Nature Biotechnology
Volume
34
Issue
3
Inclusive Pages
334-338
DOI
10.1038/nbt.3469
Keywords
CRISPR-Cas Systems--genetics; Ornithine Carbamoyltransferase--genetics; Ornithine Carbamoyltransferase Deficiency Disease--therapy; RNA Editing
APA Citation
Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., Batshaw, M. L., & Wilson, J. (2016). A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.. Nature Biotechnology, 34 (3). http://dx.doi.org/10.1038/nbt.3469
Peer Reviewed
1
Open Access
1
Comments
This is an open access PubMed Central article. Click on link for full-text access.