2020年10月
Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan.
Human gene therapy
- 巻
- 31
- 号
- 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
- ID情報
-
- DOI : 10.1089/hum.2020.156
- PubMed ID : 32731837
- PubMed Central 記事ID : PMC7585607