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NLRP3 inflammasome: From drug target to drug discovery

Ling Yin, Hongliang Zhang, Yuhua Shang, Songquan Wu, Tengchuan Jin

2025Drug Discovery Today8 citationsDOIOpen Access PDF

Abstract

The immune system employs innate and adaptive immunity to combat pathogens and stress stimuli. Innate immunity rapidly detects pathogen-associated molecular patterns (PAMPs) and damage-associated molecular patterns (DAMPs) via pattern recognition receptors (PRRs), whereas adaptive immunity mediates antigen-specific T/B cell responses. The NLRP3 inflammasome, a key cytoplasmic PRR, consists of leucine-rich repeat, nucleotide-binding, and pyrin domains. Its activation requires priming (signal 1: Toll-like receptors/NOD-like receptors/cytokine receptors) and activation (signal 2: PAMPs/DAMPs/particulates). NLRP3 triggers cytokine storms and neuroinflammation, contributing to inflammatory diseases. Emerging therapies target NLRP3 via nuclear receptors (transcriptional regulation), adeno-associated virus (AAV) vectors (gene delivery), and microRNAs (post-transcriptional modulation). This review highlights NLRP3's signaling cascade, pathological roles, and combinatorial treatments leveraging nuclear receptors, AAVs, and microRNAs for immunomodulation.

Topics & Concepts

Drug discoveryDrugInflammasomePharmacologyPharmaceutical sciencesComputational biologyMedicineChemistryBiologyBioinformaticsImmunologyInflammationInflammasome and immune disordersKawasaki Disease and Coronary Complicationsinterferon and immune responses
NLRP3 inflammasome: From drug target to drug discovery | Litcius