NACHT, LRR and PYD domains-containing protein 3 messenger RNA (NLRP3 mRNA)

NLRP3 mRNA is the transcript encoding the NACHT, LRR and PYD domains-containing protein 3, a key intracellular sensor of the innate immune system [UniProt]. The protein product assembles into the NLRP3 inflammasome, which activates caspase-1 to process pro-inflammatory cytokines IL-1β and IL-18 [PubMed]. Dysregulation of this pathway is linked to autoinflammatory diseases like Cryopyrin-Associated Periodic Syndromes (CAPS) and chronic conditions such as gout and Alzheimer's disease [NIH]. Therapeutic targeting of the mRNA using antisense oligonucleotides (ASOs) or siRNA aims to degrade the transcript and prevent protein synthesis [PubMed]. Clinical candidates like ION734 (IONIS-NLRP3-Rx) are designed to reduce NLRP3 expression to treat inflammatory disorders [Ionis Pharmaceuticals]. This approach potentially offers a more upstream intervention compared to blocking individual cytokines [PubMed].

Other names

Cryopyrin mRNACIAS1 mRNANALP3 mRNAPYPAF1 mRNACaspase recruitment domain-containing protein 7 mRNACLR1.1 mRNA

Antisense-mediated mRNA degradation via RNase H or RNA interference (RNAi) to inhibit protein translation.