A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.

Authors

Yang Yang
Lili Wang
Peter Bell
Deirdre McMenamin
Zhenning He
John White
Hongwei Yu
Chenyu Xu
Hiroki Morizono, George Washington University
Kiran Musunuru
Mark L. Batshaw, George Washington UniversityFollow
James M Wilson

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

Comments

This is an open access PubMed Central article. Click on link for full-text access.

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