The Korean journal of internal medicine

The Korean Association of Internal Medicine (대한내과학회)
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CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells

  • Kim, Eun Ji (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea) ;
  • Kang, Ki Ho (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea) ;
  • Ju, Ji Hyeon (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea)
  • Received : 2016.06.24
  • Accepted : 2016.12.10
  • Published : 2017.01.01
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Abstract

Recent advances in genome editing with programmable nucleases have opened up new avenues for multiple applications, from basic research to clinical therapy. The ease of use of the technology-and particularly clustered regularly interspaced short palindromic repeats (CRISPR)-will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. Here, we highlight the progress made in correcting gene mutations in monogenic hereditary disorders and discuss various CRISPR-associated applications, such as cancer research, synthetic biology, and gene therapy using induced pluripotent stem cells. The challenges, ethical issues, and future prospects of CRISPR-based systems for human research are also discussed.

Keywords

Acknowledgement

Supported by : National Research Foundation of Korea (NRF)

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