Complement system component mRNA

Complement system component mRNA refers to the messenger RNA transcripts that encode various proteins within the complement cascade, a vital part of the innate immune system (Janeway et al., 2001). These mRNAs, such as those for C3, C5, and Factor B, are increasingly targeted by novel therapeutic modalities like RNA interference (RNAi) and antisense oligonucleotides (ASOs) to treat diseases driven by complement overactivation (Garrelfs et al., 2022). By degrading specific mRNA transcripts, primarily in the liver, these drugs reduce the systemic production of pro-inflammatory and pore-forming complement proteins (Alnylam Pharmaceuticals, 2023). This approach is particularly relevant in conditions like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and various nephropathies where the complement system causes host tissue damage (Mastellos et al., 2019). Targeting the mRNA allows for potent and long-lasting suppression of the complement pathway compared to traditional monoclonal antibodies (Arrowhead Pharmaceuticals, 2023). However, therapeutic inhibition of these components necessitates vigilance regarding increased susceptibility to encapsulated bacterial infections, particularly Neisseria meningitidis (Hillmen et al., 2019).

Other names

Complement mRNAComplement factor mRNAComplement protein mRNA

RNA interference (RNAi) or antisense oligonucleotide (ASO) mediated degradation of specific mRNA transcripts to prevent the translation of complement proteins (Alnylam Pharmaceuticals, 2023; Ionis Pharmaceuticals, 2023).