| dc.contributor.author | Yin, Hao | |
| dc.contributor.author | Xue, Wen | |
| dc.contributor.author | Chen, Sidi | |
| dc.contributor.author | Benedetti, Eric | |
| dc.contributor.author | Grompe, Markus | |
| dc.contributor.author | Kotelianski, Victor E. | |
| dc.contributor.author | Bogorad, Roman | |
| dc.contributor.author | Sharp, Phillip A. | |
| dc.contributor.author | Jacks, Tyler E. | |
| dc.contributor.author | Anderson, Daniel Griffith | |
| dc.date.accessioned | 2015-06-05T17:19:53Z | |
| dc.date.available | 2015-06-05T17:19:53Z | |
| dc.date.issued | 2014-03 | |
| dc.date.submitted | 2013-12 | |
| dc.identifier.issn | 1087-0156 | |
| dc.identifier.issn | 1546-1696 | |
| dc.identifier.uri | http://hdl.handle.net/1721.1/97197 | |
| dc.description.abstract | We demonstrate CRISPR-Cas9–mediated correction of a Fah mutation in hepatocytes in a mouse model of the human disease hereditary tyrosinemia. Delivery of components of the CRISPR-Cas9 system by hydrodynamic injection resulted in initial expression of the wild-type Fah protein in ~1/250 liver cells. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that CRISPR-Cas9–mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases. | en_US |
| dc.description.sponsorship | National Cancer Institute (U.S.) (Grant 2-PO1-CA42063) | en_US |
| dc.description.sponsorship | National Cancer Institute (U.S.) (Core Grant P30-CA14051) | en_US |
| dc.description.sponsorship | National Institutes of Health (U.S.) (Grant R01-CA133404) | en_US |
| dc.description.sponsorship | David H. Koch Institute for Integrative Cancer Research at MIT (Marie D. and Pierre Casimir-Lambert Fund) | en_US |
| dc.description.sponsorship | National Institutes of Health (U.S.) (Centers for Cancer Nanotechnology Excellence 5-U54-CA151884-04) | en_US |
| dc.description.sponsorship | MIT-Harvard Center of Cancer Nanotechnology Excellence | en_US |
| dc.description.sponsorship | National Institutes of Health (U.S.) (1K99CA169512) | en_US |
| dc.language.iso | en_US | |
| dc.publisher | Nature Publishing Group | en_US |
| dc.relation.isversionof | http://dx.doi.org/10.1038/nbt.2884 | en_US |
| dc.rights | Article is made available in accordance with the publisher's policy and may be subject to US copyright law. Please refer to the publisher's site for terms of use. | en_US |
| dc.source | PMC | en_US |
| dc.title | Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype | en_US |
| dc.type | Article | en_US |
| dc.identifier.citation | Yin, Hao, Wen Xue, Sidi Chen, Roman L Bogorad, Eric Benedetti, Markus Grompe, Victor Koteliansky, Phillip A Sharp, Tyler Jacks, and Daniel G Anderson. “Genome Editing with Cas9 in Adult Mice Corrects a Disease Mutation and Phenotype.” Nature Biotechnology 32, no. 6 (March 30, 2014): 551–553. | en_US |
| dc.contributor.department | Institute for Medical Engineering and Science | en_US |
| dc.contributor.department | David H. Koch Institute for Integrative Cancer Research at MIT | en_US |
| dc.contributor.department | Harvard University--MIT Division of Health Sciences and Technology | en_US |
| dc.contributor.department | Massachusetts Institute of Technology. Department of Biology | en_US |
| dc.contributor.department | Massachusetts Institute of Technology. Department of Chemical Engineering | en_US |
| dc.contributor.mitauthor | Yin, Hao | en_US |
| dc.contributor.mitauthor | Xue, Wen | en_US |
| dc.contributor.mitauthor | Chen, Sidi | en_US |
| dc.contributor.mitauthor | Bogorad, Roman | en_US |
| dc.contributor.mitauthor | Sharp, Phillip A. | en_US |
| dc.contributor.mitauthor | Jacks, Tyler E. | en_US |
| dc.contributor.mitauthor | Anderson, Daniel Griffith | en_US |
| dc.relation.journal | Nature Biotechnology | en_US |
| dc.eprint.version | Author's final manuscript | en_US |
| dc.type.uri | http://purl.org/eprint/type/JournalArticle | en_US |
| eprint.status | http://purl.org/eprint/status/PeerReviewed | en_US |
| dspace.orderedauthors | Yin, Hao; Xue, Wen; Chen, Sidi; Bogorad, Roman L; Benedetti, Eric; Grompe, Markus; Koteliansky, Victor; Sharp, Phillip A; Jacks, Tyler; Anderson, Daniel G | en_US |
| dc.identifier.orcid | https://orcid.org/0000-0001-5785-8911 | |
| dc.identifier.orcid | https://orcid.org/0000-0003-1465-1691 | |
| dc.identifier.orcid | https://orcid.org/0000-0001-5629-4798 | |
| dc.identifier.orcid | https://orcid.org/0000-0001-6898-3793 | |
| mit.license | PUBLISHER_POLICY | en_US |
