miR-mask oligonucleotide

miR-mask oligonucleotide is a backbone-stabilized antisense oligonucleotide designed to selectively block the microRNA-432 (miR-432) binding site on transmembrane 6 superfamily member 2 (TM6SF2) mRNA [1036]. Developed by researchers at the University of California San Francisco (UCSF), this allele-specific therapeutic strategy targets the TM6SF2 rs58542926 (E167K) single nucleotide polymorphism (SNP) [1036]. This genetic variant introduces a novel binding site for miR-432, leading to miRNA-mediated degradation of TM6SF2 mRNA and subsequent hepatic lipid accumulation, inflammation, and fibrosis [1036]. By masking this specific binding site, the oligonucleotide prevents miR-432-mediated degradation, restoring TM6SF2 RNA and protein levels and reducing intrahepatic steatosis without altering overall miR-432 levels or disrupting native miRNA networks [1036]. It is being investigated for the treatment of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH) [1036].

Other names

TM6SF2 miR-maskTM-6SF2 miR-maskTM 6SF2 miR-maskmiR-mask