Complement component 5 (C5) mRNA (C5 mRNA)

Complement component 5 (C5) mRNA is the messenger RNA transcript responsible for the synthesis of the C5 protein, a central component of the complement system (UniProt: P01031). The complement system is a part of the innate immune response that, when overactivated, contributes to various inflammatory and autoimmune diseases (PubMed: 29107560). C5 mRNA is primarily expressed in the liver, and its translation results in the C5 protein, which is cleaved into C5a (a potent anaphylatoxin) and C5b, the latter initiating the formation of the membrane attack complex (MAC) (PubMed: 30215150). By targeting C5 mRNA using RNA interference (RNAi) or antisense technologies, the systemic production of C5 protein can be significantly reduced at the source (Alnylam Pharmaceuticals; Ionis Pharmaceuticals). This approach provides a therapeutic strategy for treating complement-mediated disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) (PubMed: 32453157). Unlike monoclonal antibodies that bind to the circulating protein, targeting the mRNA prevents the protein's synthesis, potentially offering more stable suppression of complement activity and reduced dosing frequency.

Other names

Complement C5 mRNACPAMD4 mRNAComplement component 5 messenger RNA

RNA interference (RNAi) or antisense oligonucleotide (ASO) mediated degradation of the C5 mRNA transcript, which inhibits the hepatic synthesis of the C5 protein and reduces its systemic availability (PubMed: 32453157, PubMed: 29107560).