CRISPR/Cas9: at the cutting edge of hepatology. Issue 7 (9th May 2017)

• Record Type: Journal Article
• Authors: Pankowicz, Francis P; Jarrett, Kelsey E; Lagor, William R; Bissig, Karl-Dimiter
• Is Part Of: Gut. Volume 66:Issue 7(2017)
• Page Start: 1329
• Page End: 1340
• Publication Date: 2017-05-09
• Subjects: GENE THERAPY -- GENE TARGETING -- GENE TRANSFER -- HEPATOCYTE; Gastroenterology -- Periodicals; 616.33
• DOI: 10.1136/gutjnl-2016-313565
• Languages: English
• ISSNs: 0017-5749

Abstract

Abstract : 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.