NLRP3

Chr 1AD

NLR family pyrin domain containing 3

Also known as: AGTAVPRL, AII, AVP, C1orf7, CIAS1, CLR1.1, DFNA34, FCAS

This protein functions as a sensor component of the NLRP3 inflammasome complex, which detects membrane integrity defects and triggers the secretion of inflammatory cytokines IL1B and IL18. Mutations cause a spectrum of autoinflammatory disorders including familial cold autoinflammatory syndrome, Muckle-Wells syndrome, and CINCA syndrome, as well as autosomal dominant hearing loss with or without inflammation. These conditions follow autosomal dominant inheritance and typically involve the skin, joints, central nervous system, and auditory system, with some presenting in the neonatal period.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
GOFmechanismADLOEUF 0.525 OMIM phenotypes
Clinical SummaryNLRP3
Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.33) despite low pLI — interpret in context.
📋
ClinVar Variants
4 unique Pathogenic / Likely Pathogenic· 136 VUS of 200 total submissions
💊
Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — NLRP3
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.52LOEUF
pLI 0.001
Z-score 3.94
OE 0.33 (0.210.52)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.14Z-score
OE missense 0.74 (0.690.81)
414 obs / 556.1 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.33 (0.210.52)
00.351.4
Missense OE0.74 (0.690.81)
00.61.4
Synonymous OE1.28
01.21.6
LoF obs/exp: 13 / 39.8Missense obs/exp: 414 / 556.1Syn Z: -3.41
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveNLRP3-related familial cold autoinflammatory syndromeOTHERAD
definitiveNLRP3-related chronic neurologic cutaneous and articular syndrome (CINCA)OTHERAD
strongNLRP3-related cold urticaria, familialGOFAD
DN
0.76top 25%
GOF
0.77top 25%
LOF
0.1598th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). Both the Badonyi & Marsh prediction and the broader genomic evidence point to gain-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median · 1 literature citation
DNprediction above median · 1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNThese findings strongly suggested that the mutated protein exerts a dominant-negative or a gain-of-function effect over the wildtype product and that the null mutation of 1 allele would probably have no effect or would lead to a different phenotypic expression because of haploinsufficiency.PMID:14630794
GOFThe discovery that dominant gain-of-function mutations in NLRP3 cause the cryopyrin-associated periodic syndromes (CAPS) and trigger spontaneous inflammasome activation and IL-1β oversecretion led to successful treatment with IL-1-blocking agents.PMID:25217959

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

200 submitted variants in ClinVar

Classification Summary

Pathogenic1
Likely Pathogenic3
VUS136
Likely Benign55
Benign2
Conflicting3
1
Pathogenic
3
Likely Pathogenic
136
VUS
55
Likely Benign
2
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
1
0
1
Likely Pathogenic
0
2
1
0
3
VUS
5
120
9
2
136
Likely Benign
0
2
18
35
55
Benign
1
0
1
0
2
Conflicting
3
Total61243037200

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

NLRP3 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Transgender Individuals

Effects of Gender-Affirming Hormone Therapy on Cardiovascular, Metabolic, and Mental Health Outcomes in Transgender Adults.

NOT YET RECRUITING
NCT07394400Milagros Rocha BarajasStarted 2026-02-14
Coronary Heart Disease

Adipose Tissue and Inflammation in Coronary Heart Disease

ACTIVE NOT RECRUITING
NCT02760914Oslo University HospitalStarted 2016-06
Late-Life Depression

Effects of Specific Amino Acid Supplementation and Lifestyle Factors on Brain Ageing

RECRUITING
NCT07497347Phase NAIRCCS Centro San Giovanni di Dio FatebenefratelliStarted 2026-03-01
L-serine supplementationPlacebo
Heart Failure, SystolicHeart Failure NYHA Class IVMetabolic Disturbance

Mechanistic Studies of Nicotinamide Riboside in Human Heart Failure

ACTIVE NOT RECRUITING
NCT04528004Phase EARLY_PHASE1University of WashingtonStarted 2020-09-26
Nicotinamide ribosidePlacebo
Type 2 DiabetesPrediabetes (Insulin Resistance, Impaired Glucose Tolerance)Prediabetes / Type 2 Diabetes

Effects of Different Fish Oil Types on Type 2 Diabetes Risk Factors in High-Risk Adults

NOT YET RECRUITING
NCT07575438Phase PHASE2May Faraj, PDt, PhDStarted 2026-07
Fish Oil
Type 2 DiabetesInflammationInsulin Sensitivity/Resistance

Targeting Risk Factors for Diabetes in Subjects With Normal Blood Cholesterol Using Omega-3 Fatty Acids

RECRUITING
NCT04485871Phase NAInstitut de Recherches Cliniques de MontrealStarted 2019-12-19
Omega-3 fatty acids
Neurodegenerative DiseasesCognition Disorders in Old Age

Probiotics in Mild Alzheimer's Disease

RECRUITING
NCT06181513Phase EARLY_PHASE1University of NicosiaStarted 2022-12-19
Probiotic Blend Capsule
Obstructive Sleep Apnea-Hypopnea SyndromeAnxiety DisordersDepressive Disorders

EGR2 and NLRP3 Pathways in Obstructive Sleep Apnea-Related Cognitive and Mood Disorders

RECRUITING
NCT07120711Xinhua Hospital, Shanghai Jiao Tong University School of MedicineStarted 2025-07-01
EGR2/NLRP3 pathway activityfatty tissue
Scarring Alopecia

Non-Ablative Laser to Treat Scarring Alopecia With Hair Follicle Gene Expression Analysis

RECRUITING
NCT06946550Phase NAMontefiore Medical CenterStarted 2025-12-09
1470nm non-ablative fractional laser
PeriodontitisGingivitisPeriodontal Diseases

Evaluation of the Effect of Smoking on Pyroptosis in Periodontal Pathogenesis

ACTIVE NOT RECRUITING
NCT07563192Suleyman Demirel UniversityStarted 2024-10-15
Autoinflammatory DiseaseJuvenile Dermatomyositis

Studies of the Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases Including Juvenile Dermatomyositis

RECRUITING
NCT00059748National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Started 2003-05-09
Cryopyrin Associated Periodic SyndromeFamilial Cold UrticariaMuckle-Wells Syndrome

Circadian Rhythm Deregulation in Patients With CAPS

RECRUITING
NCT06544018Phase NAHospices Civils de LyonStarted 2026-02-25
Genetic analysis of NLRP3Circadian rhythm measurementSaliva sampling
Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗