Year: 2020
Type: review
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.
Source: Human Gene Therapy
DOI: 10.1089/hum.2020.156
Institutions: Kanazawa Institute of Technology, Nihon Pharmaceutical University, National Institute of Health Sciences, The University of Tokyo, Jichi Medical University, National Center For Child Health and Development, Nippon Medical School, University of Tsukuba, Okayama University, The University of Osaka, Saitama Medical University, Pharmaceuticals and Medical Devices Agency
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
OpenAlex ID: W3046733952