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dc.contributor.authorYin, Hao
dc.contributor.authorXue, Wen
dc.contributor.authorChen, Sidi
dc.contributor.authorBenedetti, Eric
dc.contributor.authorGrompe, Markus
dc.contributor.authorKotelianski, Victor E.
dc.contributor.authorBogorad, Roman
dc.contributor.authorSharp, Phillip A.
dc.contributor.authorJacks, Tyler E.
dc.contributor.authorAnderson, Daniel Griffith
dc.date.accessioned2015-06-05T17:19:53Z
dc.date.available2015-06-05T17:19:53Z
dc.date.issued2014-03
dc.date.submitted2013-12
dc.identifier.issn1087-0156
dc.identifier.issn1546-1696
dc.identifier.urihttp://hdl.handle.net/1721.1/97197
dc.description.abstractWe 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.sponsorshipNational Cancer Institute (U.S.) (Grant 2-PO1-CA42063)en_US
dc.description.sponsorshipNational Cancer Institute (U.S.) (Core Grant P30-CA14051)en_US
dc.description.sponsorshipNational Institutes of Health (U.S.) (Grant R01-CA133404)en_US
dc.description.sponsorshipDavid H. Koch Institute for Integrative Cancer Research at MIT (Marie D. and Pierre Casimir-Lambert Fund)en_US
dc.description.sponsorshipNational Institutes of Health (U.S.) (Centers for Cancer Nanotechnology Excellence 5-U54-CA151884-04)en_US
dc.description.sponsorshipMIT-Harvard Center of Cancer Nanotechnology Excellenceen_US
dc.description.sponsorshipNational Institutes of Health (U.S.) (1K99CA169512)en_US
dc.language.isoen_US
dc.publisherNature Publishing Groupen_US
dc.relation.isversionofhttp://dx.doi.org/10.1038/nbt.2884en_US
dc.rightsArticle 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.sourcePMCen_US
dc.titleGenome editing with Cas9 in adult mice corrects a disease mutation and phenotypeen_US
dc.typeArticleen_US
dc.identifier.citationYin, 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.departmentInstitute for Medical Engineering and Scienceen_US
dc.contributor.departmentDavid H. Koch Institute for Integrative Cancer Research at MITen_US
dc.contributor.departmentHarvard University--MIT Division of Health Sciences and Technologyen_US
dc.contributor.departmentMassachusetts Institute of Technology. Department of Biologyen_US
dc.contributor.departmentMassachusetts Institute of Technology. Department of Chemical Engineeringen_US
dc.contributor.mitauthorYin, Haoen_US
dc.contributor.mitauthorXue, Wenen_US
dc.contributor.mitauthorChen, Sidien_US
dc.contributor.mitauthorBogorad, Romanen_US
dc.contributor.mitauthorSharp, Phillip A.en_US
dc.contributor.mitauthorJacks, Tyler E.en_US
dc.contributor.mitauthorAnderson, Daniel Griffithen_US
dc.relation.journalNature Biotechnologyen_US
dc.eprint.versionAuthor's final manuscripten_US
dc.type.urihttp://purl.org/eprint/type/JournalArticleen_US
eprint.statushttp://purl.org/eprint/status/PeerRevieweden_US
dspace.orderedauthorsYin, Hao; Xue, Wen; Chen, Sidi; Bogorad, Roman L; Benedetti, Eric; Grompe, Markus; Koteliansky, Victor; Sharp, Phillip A; Jacks, Tyler; Anderson, Daniel Gen_US
dc.identifier.orcidhttps://orcid.org/0000-0001-5785-8911
dc.identifier.orcidhttps://orcid.org/0000-0003-1465-1691
dc.identifier.orcidhttps://orcid.org/0000-0001-5629-4798
dc.identifier.orcidhttps://orcid.org/0000-0001-6898-3793
mit.licensePUBLISHER_POLICYen_US


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