Other

The University of Tokyo

Microbial Chemistry Research Foundation

Atsushi Nishida

Yoshikazu Takahashi

Masatoshi Hagiwara

Daishiro Ikeda

Masafumi Matsuo

Ryoichi Matsuda

Masataka Shiozuka

Yoshiaki Nonomura

Akira Wagatsuma

Naoyuki Kataoka

A readthrough inducing agent for inducing readthrough of a premature stop codon generated by nonsense mutations, the readthrough inducing agent comprising a compound having a structure expressed by the following Structural Formula (A), and a drug for treating a genetic disease caused by nonsense mutations, the drug comprising the readthrough inducing agent. 

Readthrough inducing agent and drug for treating genetic disease caused by nonsense mutation

An object of the present invention is to provide a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation. The prophylactic or ameliorating agent used in the present invention is a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation, wherein the prophylactic or ameliorating agent contains a compound having a molecular weight of 1500 or lower.

Prophylactic or ameliorating agent for genetic diseases

Growing community of inventors

Hyogo, Japan

Atsushi Nishida