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dc.contributor.authorSchneller, Jessica L
Lee, Ciaran M
Bao, Gang
Venditti, Charles P
dc.date.accessioned 2017-02-27T07:02:21Z
dc.date.available 2017-02-27T07:02:21Z
dc.date.issued 2017-02-27
dc.identifier.citation Schneller, Jessica L, Lee, Ciaran M, Bao, Gang, et al.. "Genome editing for inborn errors of metabolism: advancing towards the clinic." BMC Medicine, 15, no. 1 (2017) http://dx.doi.org/10.1186/s12916-017-0798-4.
dc.identifier.urihttps://hdl.handle.net/1911/94012
dc.description.abstract Abstract Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative. Advances in the field of genome editing have recently resulted in the in vivo correction of murine models of IEM. Site-specific endonucleases, such as zinc-finger nucleases and the CRISPR/Cas9 system, in combination with delivery vectors engineered to target disease tissue, have enabled correction of mutations in disease models of hemophilia B, hereditary tyrosinemia type I, ornithine transcarbamylase deficiency, and lysosomal storage disorders. These in vivo gene correction studies, as well as an overview of genome editing and future directions for the field, are reviewed and discussed herein.
dc.rightsThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/
dc.title Genome editing for inborn errors of metabolism: advancing towards the clinic
dc.type Journal article
dc.citation.journalTitle BMC Medicine
dc.citation.volumeNumber 15
dc.citation.issueNumber 1
dc.contributor.publisher BioMed Central
dc.date.updated 2017-02-27T07:02:21Z
dc.type.dcmi Text
dc.identifier.doihttp://dx.doi.org/10.1186/s12916-017-0798-4
dc.language.rfc3066 en
dc.type.publication publisher version
dcterms.bibliographicCitation BMC Medicine. 2017 Feb 27;15(1):43
dc.rights.holder The Author(s).
local.sword.agent BioMed Central
dc.citation.articleNumber 43


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This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
Except where otherwise noted, this item's license is described as This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.