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Open Access
  • © Teruhide Yamaguchi
  • , et al.
  • 2022

Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan

  • Teruhide Yamaguchi 1
  • Eriko Uchida 2
  • Takashi Okada 3
  • Keiya Ozawa 4
  • Masafumi Onodera 5
  • Akihiro Kume 4
  • Takashi Shimada 6
  • Satoru Takahashi 7
  • Kenzaburo Tani 3
  • Yasutomo Nasu 8
  • Tomoji Mashimo 3
  • Hiroyuki Mizuguchi 9
  • Kohnosuke Mitani 10
  • Kazushige Maki 11
  • 1 - Kanazawa Institute of Technology - Ishikawa Japan
  • 2 - National Institute of Health Sciences
  • 3 - The University of Tokyo
  • 4 - Jichi Medical University
  • 5 - National Center for Child Health and Development
  • 6 - Nippon Medical School
  • 7 - University of Tsukuba
  • 8 - Okayama University Graduate School of Medicine - Dentistry and Pharmaceutical Sciences
  • 9 - Osaka University
  • 10 - Saitama Medical University
  • 11 - Pharmaceuticals and Medical Devices Agency

Abstract

The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinical development using these genome-editing tools is progressing rapidly. However, it is also becoming clear that there is a possibility of unintended gene sequence modification or deletion, or the insertion of undesired genes, or the selection of cells with abnormalities in the cancer suppressor gene p53; these unwanted actions are not possible with current gene therapy. The Science Board of the Pharmaceuticals and Medical Devices Agency of Japan has compiled a report on the expected aspects of such genome-editing technology and the risks associated with it. This article summarizes the history of that discussion and compares the key concepts with information provided by other regulatory authorities.