
- © Akihiro Kume
- , et al.
- 2022
Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan
- Akihiro Kume 1
- Eriko Uchida 2
- Hiroyuki Mizuguchi 3
- Kazushige Maki 4
- Keiya Ozawa 1
- Kenzaburo Tani 5
- Kohnosuke Mitani 6
- Masafumi Onodera 7
- Satoru Takahashi 8
- Takashi Okada 5
- Takashi Shimada 9
- Teruhide Yamaguchi 10
- Tomoji Mashimo 5
- Yasutomo Nasu 11
- 1 - Jichi Medical University
- 2 - National Institute of Health Sciences
- 3 - Osaka University
- 4 - Pharmaceuticals and Medical Devices Agency
- 5 - The University of Tokyo
- 6 - Saitama Medical University
- 7 - National Center for Child Health and Development
- 8 - University of Tsukuba
- 9 - Nippon Medical School
- 10 - Kanazawa Institute of Technology - Ishikawa Japan
- 11 - Okayama University Graduate School of Medicine - Dentistry and Pharmaceutical Sciences
Jun 24, 2022
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.
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