PHARMACEUTICAL COMPOSITION AND TREATMENT METHOD FOR GENETIC DISEASE ASSOCIATED WITH SPLICING ABNORMALITIES
申请人:KYOTO UNIVERSITY
公开号:US20200113907A1
公开(公告)日:2020-04-16
A pharmaceutical composition for genetic diseases caused by an aberrant splicing regulation is provided.
Provided are a pharmaceutical composition for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases caused by an aberrant splicing regulation, the pharmaceutical composition containing, as an active ingredient, a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation, and a method for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases using a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation.