anti-mir-128-3p antisense oligonucleotide

anti-mir-128-3p antisense oligonucleotide is an RNA-based therapeutic designed to inhibit the pathological microRNA miR-128-3p. In the context of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), miR-128-3p levels are elevated following the loss of dystrophin, leading to impaired mitochondrial function, muscle wasting, and cardiac dysfunction. By sequestering miR-128-3p, this antisense oligonucleotide (ASO) restores the expression of critical metabolic regulators such as PGC-1a, SIRT1, and PPARa. Preclinical evidence in mdx5cv mice and DMDY/- pigs indicates that targeting miR-128-3p can mitigate muscle weakness, improve mitochondrial health, and significantly ameliorate cardiac insufficiency, including improvements in left ventricular ejection fraction.

Other names

anti-miR-128-3p ASOanti-miR128-3p ASOanti-miR 128-3p ASO