MicroRNA-206 Downregulation Improves Therapeutic Gene Expression and Motor Function in mdx Mice.

ABSTRACT: Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by a mutation in the dystrophin gene. Numerous gene therapies have been developed to replace or repair the defective dystrophin gene; however, these treatments cannot restore the full-length protein or completely resolve dystrophic symptoms. Secondary pathological mechanisms, such as functional ischemia and fibrosis, are thought to exacerbate the primary defect and cause the profound muscle degeneration found in dystrophic muscle. Surrogate therapies utilizing alternative therapeutic genes, or "booster genes," such as VEGFA and utrophin, seek to address these secondary mechanisms and have shown impressive benefit in mdx mice. A skeletal muscle-specific microRNA, miR-206, is particularly overexpressed in dystrophic

SUBMITTER: Bulaklak K 

PROVIDER: S-EPMC6011022 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature