UTROPHIN UPREGULATION COMPOUNDS FOR DUCHENNE MUSCULAR DYSTROPHY THERAPY

摘要

The present invention provides methods for increasing expression of utrophin in a subject in need thereof, the method comprising administering to the subject a pharmaceutical composition comprising a post-transcriptionally utrophin upregulator compound. The present invention also provides methods for treating a subject having a muscular dystrophy. This invention further provides methods for high-throughput screening for a post-transcriptional utrophin upregulator compound.