Current issues on reflection paper for genome editing gene therapy

• YAMAGUCHI Teruhide

  Kanazawa-Institute of Technology Nihon Pharmaceutical University

Bibliographic Information

Other Title

• ゲノム編集に関するPMDA科学委員会コンセプトペーパーについて

Abstract

In a past decade, advances in genome editing technologies have provided a possibility ofa novel gene therapy not only in vitro but also in vivo. The genome editing is the technology to cause either the knock-out of target gene or modification of abnormal gene by homologous recombination of target gene (HDR) through the double strand break of target DNA sequence. The targeting ability of genome editing is dependent on the recognition by specific protein to be able to bind target DNA sequence (Zinc-Finger nuclease; ZFN or transcription activator-like effector nuclease ; TALEN) or binding of a single-guide RNA (sgRNA)of which form the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR associated proteins (Cas) complex. Many clinical study protocols using genome editing tools have been approved and been ongoing.Advantageous characters of genome editing are utilized to exploit either the knockout technology for thetarget gene by HNEJ or modification of hereditary abnormal genewith normal one by HDR. On the other hand, genome editing technologies have become known to cause DSB on un-target gene (off-target effect), or yield insertions and deletions, or template repair from a separate donor DNA molecule (on-target mutation). Therefore, to overcome the undesirable effects of genome editing should be continuing to develop the analytical methods or new technology. Science Board in PMDA has established the Committee for Genome Editing to discuss about the quality and safety issues of technologies. In this meeting I would like to discuss to refer this paper.

Journal

• Annual Meeting of the Japanese Society of Toxicology

  Annual Meeting of the Japanese Society of Toxicology 47.1 (0), S15-1-, 2020

  The Japanese Society of Toxicology

Details

• CRID: 1390848647545190400
• NII Article ID: 130007898527
• DOI: 10.14869/toxpt.47.1.0_s15-1
• Text Lang: ja
• Data Source:
  • JaLC
  • CiNii Articles
• Abstract License Flag: Disallowed