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.
- GENE THERAPY
- GENE TARGETING
- GENE TRANSFER
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Contributors All authors have contributed, written and accepted final version of the manuscript.
Funding National Heart and Lung Institute (HL132840, R01HL134510, T32HL07676 and T32HL092332) and Cancer Prevention and Research Institute of Texas (RP150587).
Competing interests None declared.
Provenance and peer review Commissioned; internally peer reviewed.
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