Mannan-binding lectin-associated serine protease 2 mRNA (MASP-2 mRNA)

Mannan-binding lectin-associated serine protease 2 (MASP-2) mRNA is the transcript of the MASP2 gene, which encodes a key serine protease essential for the activation of the lectin pathway of the complement system (UniProt P48740). MASP-2 is primarily synthesized in the liver and, upon secretion into the blood, associates with pattern recognition molecules to initiate a proteolytic cascade that leads to opsonization and inflammation (PubMed: 31530574). Dysregulation or overactivation of the lectin pathway is a significant driver in the pathogenesis of several rare and severe diseases, including IgA nephropathy, atypical hemolytic uremic syndrome (aHUS), and various thrombotic microangiopathies (PubMed: 33159013). Therapeutic strategies targeting MASP-2 mRNA, such as the antisense oligonucleotide IONIS-MASP2-LRx (also known as ION959), utilize RNase H-mediated degradation to reduce the hepatic production of the MASP-2 protein (Ionis Pharmaceuticals). By lowering systemic MASP-2 levels, these therapies aim to selectively inhibit the lectin pathway while leaving the classical and alternative complement pathways intact. This approach potentially offers a more targeted safety profile compared to broad complement inhibitors by sparing other arms of the innate immune response.

Other names

MASP2 mRNAMannose-binding lectin-associated serine protease 2 mRNAsMAP mRNAMAP19 mRNA

Antisense oligonucleotide-mediated degradation of mRNA to inhibit the translation of MASP-2 protein.