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

• Yamaguchi, Teruhide

• Uchida Eriko

• Okada, Takashi

• Ozawa, Keiya

• Onodera, Masafumi

• Kume, Akihiro

• Shimada, Takashi

• TAKAHASHI, Satoru

• Tani, Kenzaburo

• Nasu, Yasutomo

• Mashimo, Tomoji

• Mizuguchi, Hiroyuki

• Mitani, Kohnosuke

• Maki, Kazushige

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.

Journal

• Human Gene Therapy

  Human Gene Therapy 31 (19-20), 1043-1053, 2020-10

  Mary Ann Liebert

Keywords

• Genetics
• Molecular Biology
• Molecular Medicine

Details

• CRID: 1050007934538468480
• NII Article ID: 120007147867
• NII Book ID: AA10805126
• DOI: 10.1089/hum.2020.156
• ISSN: 15577422
• ISSN: 10430342
• HANDLE: 2241/0002001263
• Web Site: https://tsukuba.repo.nii.ac.jp/records/2001263
• Web Site: https://www.liebertpub.com/doi/full-xml/10.1089/hum.2020.156
• Web Site: https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.156
• Text Lang: en
• Article Type: journal article
• Data Source:
  • IRDB
  • Crossref
  • CiNii Articles
  • KAKEN