PROPHYLACTIC OR AMELIORATING AGENT FOR GENETIC DISEASES

摘要

Disclosed is a prophylactic or ameliorating agent for a genetic disease which is induced by the mutation of an exon in a gene and in which an exon that contains the mutation is skipped so as to produce a functional truncated protein. Specifically disclosed is a prophylactic or ameliorating agent for a genetic disease which is induced by the mutation of an exon in a gene and in which an exon that contains the mutation is skipped so as to produce a functional truncated protein, wherein the prophylactic or ameliorating agent is characterized by comprising a compound having a molecular weight of 1500 or less.