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

Teruhide Yamaguchi, Eriko Uchida, Takashi Okada, Keiya Ozawa, Masafumi Onodera, Akihiro Kume, Takashi Shimada, Satoru Takahashi, Kenzaburo Tani, Yasutomo Nasu, Tomoji Mashimo, Hiroyuki Mizuguchi, Kohnosuke Mitani, Kazushige Maki

Research output: Contribution to journalReview articlepeer-review

6 Citations (Scopus)


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 genomeediting 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.

Original languageEnglish
Pages (from-to)1043-1053
Number of pages11
JournalHuman Gene Therapy
Issue number19-20
Publication statusPublished - Oct 2020


  • Double-strand break
  • Gene therapy
  • Genome editing
  • Off-target effect
  • On-target mutagenesis
  • Safety

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics


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