CRISPR/Cas9: at the cutting edge of hepatology

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome engineering has revolutionised biomedical science and we are standing on the cusp of medical transformation. The therapeutic potential of this technology is tremendous, however, its translation to the clinic will be chall...

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Bibliographic Details
Published inGut Vol. 66; no. 7; pp. 1329 - 1340
Main Authors Pankowicz, Francis P, Jarrett, Kelsey E, Lagor, William R, Bissig, Karl-Dimiter
Format Journal Article Book Review
LanguageEnglish
Published England BMJ Publishing Group LTD 01.07.2017
Subjects
Animals
Clustered Regularly Interspaced Short Palindromic Repeats - genetics
CRISPR-Cas Systems
Gene Editing
Gene Targeting
Gene Transfer Techniques
Genetic Therapy
Hepatitis B virus - genetics
Humans
Mutagenesis
Mutation
Recombinational DNA Repair
GENE TRANSFER
GENE THERAPY
HEPATOCYTE
GENE TARGETING
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Summary:Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome engineering has revolutionised biomedical science and we are standing on the cusp of medical transformation. The therapeutic potential of this technology is tremendous, however, its translation to the clinic will be challenging. In this article, we review recent progress using this genome editing technology and explore its potential uses in studying and treating diseases of the liver. We discuss the development of new research tools and animal models as well as potential clinical applications, strategies and challenges.
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ISSN:0017-5749
1468-3288
DOI:10.1136/gutjnl-2016-313565