anti-miR-128-1 antisense oligonucleotide

Anti-miR-128-1 antisense oligonucleotide is a locked nucleic acid (LNA) modified therapeutic designed to inhibit miR-128-1, a microRNA that acts as a pathological modifier in Duchenne muscular dystrophy (DMD). miR-128-1 is a negative regulator of several key metabolic and mitochondrial genes, including PPARs, PGC1a, AMPK, and SIRT1. In DMD patients and animal models, miR-128-1 levels are elevated, contributing to mitochondrial dysfunction, muscle necrosis, and fibrosis. By sequestering miR-128-1, this antisense oligonucleotide de-represses these metabolic targets, restoring mitochondrial function and significantly ameliorating the pathological manifestations of dystrophin loss. Preclinical studies have demonstrated its efficacy in rescuing grip strength, treadmill endurance, and cardiac function in mouse, dog, and pig models of DMD.

Other names

anti-miR-128-1 LNA ASOanti-miR128-1 LNA ASOanti-miR 128-1 LNA ASOmiR-128-1 inhibitormiR128-1 inhibitormiR 128-1 inhibitoranti-miR-128-1anti-miR128-1anti-miR 128-1