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dc.contributor.authorEid, Ayman
dc.contributor.authorMahfouz, Magdy M.
dc.date.accessioned2017-01-30T13:02:47Z
dc.date.available2017-01-30T13:02:47Z
dc.date.issued2016-10-14
dc.identifier.citationEid A, Mahfouz MM (2016) Genome editing: the road of CRISPR/Cas9 from bench to clinic. Experimental & Molecular Medicine 48: e265. Available: http://dx.doi.org/10.1038/emm.2016.111.
dc.identifier.issn2092-6413
dc.identifier.doi10.1038/emm.2016.111
dc.identifier.urihttp://hdl.handle.net/10754/622818
dc.description.abstractMolecular scissors engineered for site-specific modification of the genome hold great promise for effective functional analyses of genes, genomes and epigenomes and could improve our understanding of the molecular underpinnings of disease states and facilitate novel therapeutic applications. Several platforms for molecular scissors that enable targeted genome engineering have been developed, including zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and, most recently, clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated-9 (Cas9). The CRISPR/Cas9 system's simplicity, facile engineering and amenability to multiplexing make it the system of choice for many applications. CRISPR/Cas9 has been used to generate disease models to study genetic diseases. Improvements are urgently needed for various aspects of the CRISPR/Cas9 system, including the system's precision, delivery and control over the outcome of the repair process. Here, we discuss the current status of genome engineering and its implications for the future of biological research and gene therapy.
dc.description.sponsorshipWe thank members of the genome-engineering laboratory at KAUST for discussions. Research in the MMM laboratory for genome engineering is supported by King Abdullah University of Science and Technology.
dc.publisherSpringer Nature
dc.relation.urlhttp://www.nature.com/emm/journal/v48/n10/full/emm2016111a.html
dc.rightsThis work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/.
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.titleGenome editing: the road of CRISPR/Cas9 from bench to clinic
dc.typeArticle
dc.contributor.departmentBiological and Environmental Sciences and Engineering (BESE) Division
dc.contributor.departmentBioscience Program
dc.contributor.departmentDesert Agriculture Initiative
dc.contributor.departmentLaboratory for Genome Engineering
dc.contributor.departmentPlant Science
dc.contributor.departmentPlant Science Program
dc.identifier.journalExperimental & Molecular Medicine
dc.eprint.versionPublisher's Version/PDF
kaust.personEid, Ayman
kaust.personMahfouz, Magdy M.
refterms.dateFOA2018-06-14T05:56:24Z
dc.date.published-online2016-10-14
dc.date.published-print2016-10


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This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/.
Except where otherwise noted, this item's license is described as This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/.