Dynamin 2 (DNM2) mRNA (DNM2 mRNA)

Dynamin 2 (DNM2) mRNA encodes a ubiquitously expressed GTPase that is essential for membrane remodeling and endocytosis. It is primarily responsible for the scission of clathrin-coated vesicles from the plasma membrane and also regulates actin cytoskeleton dynamics and centrosome cohesion (UniProt P50570). Mutations in the DNM2 gene are a primary cause of autosomal dominant centronuclear myopathy (CNM) and Charcot-Marie-Tooth disease (PubMed: 16244673). Additionally, DNM2 has been identified as a modifier in X-linked myotubular myopathy (XLMTM); reducing DNM2 levels has been shown to rescue the disease phenotype in animal models by restoring the balance of phosphoinositide metabolism (PubMed: 28262751). Consequently, DNM2 mRNA is targeted by antisense oligonucleotides (ASOs) like DYN101, which aim to reduce the overabundance or toxic activity of the protein by promoting the degradation of its mRNA transcript (ClinicalTrials.gov: NCT04033159). This therapeutic approach seeks to restore cellular homeostasis in muscle tissues where DNM2 activity is pathologically elevated or dysregulated. While clinical development of some DNM2-targeted ASOs has faced strategic pauses, the mRNA remains a significant target for genetic therapies in rare neuromuscular diseases.

Other names

DNM2DYN2Dynamin-2 mRNACentronuclear myopathy protein 2 mRNA

Antisense oligonucleotide-mediated degradation of mRNA via RNase H1 recruitment to reduce protein expression.