MicroRNA-33 inhibition ameliorates muscular dystrophy in mice

Progressive weakness and degeneration of skeletal muscles are characteristics of muscular dystrophy.

In this study the researchers identified miR-33a/b as negative regulators of skeletal muscle regeneration by targeting Cdk6, Fst, and Abca1.

Knocking out miR-33a accelerated muscle regeneration and ameliorated the dystrophic phenotype in mdx mice and knock-in of miR-33b exacerbated muscle pathology in mdx mice.

The authors identified Cdk6, Fst, and Abca1 as direct targets of miR-33a/b involved in muscle regeneration.

Antisense inhibition of miR-33a/b improved dystrophic phenotype in mdx mice and upregulated target genes in myotube from DMD patient-derived iPSCs.

They show that inhibition of miR-33a/b can be a promising strategy for the treatment of muscular dystrophy including DMD.

https://www.embopress.org/doi/full/10.1038/s44321-025-00273-9

https://sciencemission.com/MicroRNA-33-inhibition-ameliorates-muscular-dystrophy