Format

Send to

Choose Destination
Gut. 2017 Jul;66(7):1329-1340. doi: 10.1136/gutjnl-2016-313565. Epub 2017 May 9.

CRISPR/Cas9: at the cutting edge of hepatology.

Pankowicz FP1,2, Jarrett KE3,4, Lagor WR1,3,4,5, Bissig KD1,2,5,6,7,8,9.

Author information

1
Center for Cell and Gene Therapy, Center for Stem Cells and Regenerative Medicine, Baylor College of Medicine, Houston, Texas, USA.
2
Graduate Program Department of Molecular & Cellular Biology, Baylor College of Medicine, Houston, Texas, USA.
3
Department of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, Texas, USA.
4
Integrative Molecular and Biomedical Sciences Graduate Program, Baylor College of Medicine, Houston, Texas, USA.
5
Texas Medical Center Digestive Diseases Center, Baylor College of Medicine, Houston, Texas, USA.
6
Graduate Program in Translational Biology and Molecular Medicine, Baylor College of Medicine, Houston, Texas, USA.
7
Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, Texas, USA.
8
Program in Developmental Biology, Baylor College of Medicine, Houston, Texas, USA.
9
Dan L. Duncan Cancer Center, Baylor College of Medicine, Houston, Texas, USA.

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.

KEYWORDS:

GENE TARGETING; GENE THERAPY; GENE TRANSFER; HEPATOCYTE

PMID:
28487442
PMCID:
PMC5878048
DOI:
10.1136/gutjnl-2016-313565
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for HighWire Icon for PubMed Central
Loading ...
Support Center