Genome editing: the road of CRISPR/Cas9 from bench to clinic

Handle URI:
http://hdl.handle.net/10754/622818
Title:
Genome editing: the road of CRISPR/Cas9 from bench to clinic
Authors:
Eid, Ayman; Mahfouz, Magdy M. ( 0000-0002-0616-6365 )
Abstract:
Molecular 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.
KAUST Department:
Laboratory for Genome Engineering, Division of Environmental and Biological Sciences and Engineering, King Abdullah University of Science and Technology, Thuwal, Saudi Arabia.
Citation:
Eid 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.
Publisher:
Springer Nature
Journal:
Experimental & Molecular Medicine
Issue Date:
14-Oct-2016
DOI:
10.1038/emm.2016.111
Type:
Article
ISSN:
2092-6413
Sponsors:
We 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.
Additional Links:
http://www.nature.com/emm/journal/v48/n10/full/emm2016111a.html
Appears in Collections:
Articles

Full metadata record

DC FieldValue Language
dc.contributor.authorEid, Aymanen
dc.contributor.authorMahfouz, Magdy M.en
dc.date.accessioned2017-01-30T13:02:47Z-
dc.date.available2017-01-30T13:02:47Z-
dc.date.issued2016-10-14en
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.en
dc.identifier.issn2092-6413en
dc.identifier.doi10.1038/emm.2016.111en
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.en
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.en
dc.publisherSpringer Natureen
dc.relation.urlhttp://www.nature.com/emm/journal/v48/n10/full/emm2016111a.htmlen
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/.en
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/en
dc.titleGenome editing: the road of CRISPR/Cas9 from bench to clinicen
dc.typeArticleen
dc.contributor.departmentLaboratory for Genome Engineering, Division of Environmental and Biological Sciences and Engineering, King Abdullah University of Science and Technology, Thuwal, Saudi Arabia.en
dc.identifier.journalExperimental & Molecular Medicineen
dc.eprint.versionPublisher's Version/PDFen
kaust.authorEid, Aymanen
kaust.authorMahfouz, Magdy M.en
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