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CRISPR/Cas9: at the cutting edge of hepatology
  1. Francis P Pankowicz1,2,
  2. Kelsey E Jarrett3,4,
  3. William R Lagor1,3,4,5,
  4. Karl-Dimiter Bissig1,2,5,6,7,8,9
  1. 1Center for Cell and Gene Therapy, Center for Stem Cells and Regenerative Medicine, Baylor College of Medicine, Houston, Texas, USA
  2. 2Graduate Program Department of Molecular & Cellular Biology, Baylor College of Medicine, Houston, Texas, USA
  3. 3Department of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, Texas, USA
  4. 4Integrative Molecular and Biomedical Sciences Graduate Program, Baylor College of Medicine, Houston, Texas, USA
  5. 5Texas Medical Center Digestive Diseases Center, Baylor College of Medicine, Houston, Texas, USA
  6. 6Graduate Program in Translational Biology and Molecular Medicine, Baylor College of Medicine, Houston, Texas, USA
  7. 7Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, Texas, USA
  8. 8Program in Developmental Biology, Baylor College of Medicine, Houston, Texas, USA
  9. 9Dan L. Duncan Cancer Center, Baylor College of Medicine, Houston, Texas, USA
  1. Correspondence to Dr Karl-Dimiter Bissig, Department of Molecular and Cellular Biology, Baylor College of Medicine, 1, Baylor Plaza, Houston, TX 77030-3411, USA; bissig{at}bcm.edu

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.

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Footnotes

  • 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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