Methods of screening for compounds for treating muscular dystrophy using mIGF1 mRNA translation regulation

摘要

The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular mystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).