論文

国際誌
2020年10月

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

Human gene therapy
  • 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
  • 全て表示

31
19-20
開始ページ
1043
終了ページ
1053
記述言語
英語
掲載種別
研究論文(学術雑誌)
DOI
10.1089/hum.2020.156

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.

リンク情報
DOI
https://doi.org/10.1089/hum.2020.156
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/32731837
PubMed Central
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7585607
ID情報
  • DOI : 10.1089/hum.2020.156
  • PubMed ID : 32731837
  • PubMed Central 記事ID : PMC7585607

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