Plasma protease C1 inhibitor mRNA (SERPING1 mRNA)

SERPING1 mRNA encodes the plasma protease C1 inhibitor (C1-INH), a critical regulatory protein belonging to the serpin superfamily (UniProt P05155). It functions as the primary inhibitor of the C1 complex in the classical complement pathway and regulates the kinin-kallikrein and intrinsic coagulation systems by inactivating proteases like plasma kallikrein and factor XIIa (PubMed PMID: 31509094). Deficiencies or malfunctions in this protein, caused by mutations in the SERPING1 gene, result in Hereditary Angioedema (HAE), a genetic disorder marked by unpredictable and potentially fatal episodes of swelling (NIH MedlinePlus). In the context of modern therapeutics, SERPING1 mRNA is utilized as a target for mRNA replacement technology, such as Moderna's mRNA-3930 (Moderna Pipeline). This approach uses lipid nanoparticles to deliver the genetic instructions for C1-INH directly to the liver to achieve sustained endogenous production of the functional protein. By restoring inhibitor levels, the treatment aims to normalize bradykinin levels and prevent the vascular permeability associated with HAE attacks.

Other names

C1 esterase inhibitor mRNAC1-INH mRNASerpin family G member 1 mRNAC1INH mRNA

mRNA replacement therapy providing the genetic template for endogenous synthesis of functional C1 inhibitor protein to restore regulation of the complement and kinin-kallikrein pathways (Moderna Pipeline).