Genome editing for inborn errors of metabolism: advancing towards the clinic
| dc.citation.articleNumber | 43 | |
| dc.citation.issueNumber | 1 | |
| dc.citation.journalTitle | BMC Medicine | |
| dc.citation.volumeNumber | 15 | |
| dc.contributor.author | Schneller, Jessica L | |
| dc.contributor.author | Lee, Ciaran M | |
| dc.contributor.author | Bao, Gang | |
| dc.contributor.author | Venditti, Charles P | |
| dc.date.accessioned | 2017-02-27T07:02:21Z | |
| dc.date.available | 2017-02-27T07:02:21Z | |
| dc.date.issued | 2/27/2017 | |
| dc.date.updated | 2017-02-27T07:02:21Z | |
| dc.description.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.identifier.citation | Schneller, Jessica L, Lee, Ciaran M, Bao, Gang, et al.. "Genome editing for inborn errors of metabolism: advancing towards the clinic." <i>BMC Medicine,</i> 15, no. 1 (2017) BioMed Central: http://dx.doi.org/10.1186/s12916-017-0798-4. | |
| dc.identifier.doi | http://dx.doi.org/10.1186/s12916-017-0798-4 | |
| dc.identifier.uri | https://hdl.handle.net/1911/94012 | |
| dc.language.iso | eng | |
| dc.publisher | BioMed Central | |
| dc.rights | 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. | |
| dc.rights.uri | https://creativecommons.org/licenses/by/4.0/ | |
| dc.title | Genome editing for inborn errors of metabolism: advancing towards the clinic | |
| dc.type | Journal article | |
| dc.type.dcmi | Text | |
| dc.type.publication | publisher version | |
| local.sword.agent | BioMed Central |