antimir-23b-v1

Antimir-23b-v1 is an antisense oligonucleotide therapy designed to selectively inhibit microRNA miR-23b. The inhibition of miR-23b leads to upregulation of the muscleblind-like splicing regulator protein MBNL1, which is depleted in myotonic dystrophy type 1 (DM1) due to both mutant DMPK RNA sequestration and miR-23b–mediated repression. By blocking miR-23b, antimir-23b-v1 restores MBNL1 expression, corrects alternative splicing defects, and rescues myogenic differentiation parameters in DM1 cells and mouse models. The compound typically uses a chemically modified antisense scaffold and is administered either subcutaneously or intravenously in preclinical studies. Antimir-23b-v1 showed dose-dependent reductions of miR-23b, upregulation of MBNL1, and functional improvements such as increased grip strength and correction of myotonia in mouse models. Later-generation versions (V2) use an improved backbone and conjugation chemistry for higher potency and safety, but antimir-23b-v1 demonstrated proof of concept for this mechanism[1][2][3].

Other names

antimiR-23b-V1antimiR23b-V1antimiR 23b-V1