Development of antisense oligonucleotides that enable highly efficient RNA editing at pathogenic point mutations

Principal Investigator

Yukio Kawahara
Professor, Osaka University Graduate School of Medicine
Although many neurological and psychiatric diseases are still difficult to treat, some of these diseases, which are caused by single nucleotide mutations (point mutations) in the responsible genes, are treatable by correcting these mutations. However, there are various technical and ethical barriers to modifying the genome. On the other hand, modification at the RNA level is reversible and has relatively low barriers. For this reason, this project aims to establish a method for designing antisense nucleic acids (ASOs) that can efficiently edit the point mutations to the wild type at the RNA level for the development of treatments for neurological and psychiatric disorders caused by specific genetic point mutations.

Co-Investigator

Yuki Kato
Associate Professor, Osaka University Graduate School of Medicine
Taisuke Nakahama
Assistant Professor, Osaka University Graduate School of Medicine