Sat. Mar 30, 2024 9:45 AM - 11:45 AM [Room 315] Conference Center 315 (3F)

Organizer: Tomohito Tsukamoto (Grad. Sch. Pharm. Sci., Osaka Univ.), Daisuke Matsumoto (Grad. Sch. of Biomed. and Health Sci., Hiroshima Univ.)

CRISPR -Cas9 system, developed on the basis of prokaryotic acquired immunity mechanisms, can cleave targeted genomic regions with high specificity, since gRNAs (guide RNAs) designed into arbitrary genomic regions guide Cas9, which possesses DNA cleavage activity, into the genome. This property makes it possible to knock-out or knock-in any gene, which is an essential technique not only in molecular biology research but also for the construction of various disease model cells and drug screening systems. Gene therapy based on genome editing technology is expected to lead to a revolution in gene therapy, since it enables the insertion of genes anywhere in the genome and the direct repair of gene mutations. However, targeted diseases of genome editing gene therapy are limited to a few that are relatively easy to treat. Therefore, various issues remain to be resolved in the advancement of gene therapy using genome editing, including genome editing efficiency, Cas9 control methods, tissue delivery methods and patent issues.
In this symposium, five researchers working on a variety of issues related to genome editing present a wide range of their latest research, from the development of basic genome editing technology to research aimed at therapeutic applications. We would like to discuss the current stage of genome-edited therapies and the issues to be overcome in this symposium. We hope that this symposium contributes to development of novel genome-editing studies.