NLRP1

Chr 17ARAD

NLR family pyrin domain containing 1

Also known as: AIADK, CARD7, CIDED, CLR17.1, DEFCAP, DEFCAP-L/S, JRRP, MSPC

The NLRP1 protein acts as a sensor component of the NLRP1 inflammasome, recognizing pathogen-associated signals and damage signals to initiate inflammatory responses and pyroptosis through caspase-1 activation. Mutations cause autoinflammation with arthritis and dyskeratosis, palmoplantar carcinoma with multiple self-healing lesions, and juvenile recurrent respiratory papillomatosis, with both autosomal dominant and autosomal recessive inheritance patterns reported. The gene shows low constraint against loss-of-function variants, suggesting tolerance to protein-truncating mutations.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismAR/ADLOEUF 0.844 OMIM phenotypes
Clinical SummaryNLRP1
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
4 unique Pathogenic / Likely Pathogenic· 297 VUS of 500 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.84LOEUF
pLI 0.000
Z-score 2.60
OE 0.64 (0.490.84)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
1.49Z-score
OE missense 0.85 (0.800.91)
685 obs / 803.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.64 (0.490.84)
00.351.4
Missense OE0.85 (0.800.91)
00.61.4
Synonymous OE0.96
01.21.6
LoF obs/exp: 39 / 60.9Missense obs/exp: 685 / 803.8Syn Z: 0.62
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongNLRP1-related autoinflammation with arthritis and dyskeratosisOTHERAR
limitedNLRP1-related corneal intraepithelial dyskeratosisOTHERAD
DN
0.78top 25%
GOF
0.6833th %ile
LOF
0.1697th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). The Badonyi & Marsh model scores dominant-negative highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports gain-of-function. 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

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

GOFMoreover, gain-of-function mutations of NLRP1 cause inflammatory skin syndromes and a predisposition for the development of skin cancer.PMID:32640751

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

ClinVar Variant Classifications

500 submitted variants in ClinVar

Classification Summary

Pathogenic4
VUS297
Likely Benign171
Benign1
Conflicting3
4
Pathogenic
297
VUS
171
Likely Benign
1
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
4
0
4
Likely Pathogenic
0
0
0
0
0
VUS
25
253
19
0
297
Likely Benign
0
1
44
126
171
Benign
0
0
1
0
1
Conflicting
3
Total2525468126476

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

View in ClinVar →

Protein Context — Lollipop Plot

NLRP1 · 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

No active trials found for this gene.

Search ClinicalTrials.gov →
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 ↗