Information on NLRP3
Basic details
Alt. symbols: C1orf7 | CIAS1 | DFNA34 | AGTAVPRL | AII | AVP | FCAS | FCU | NALP3 | PYPAF1 | MWS | CLR1.1
Approved name: NLR family pyrin domain containing 3
Alt. names: cold autoinflammatory syndrome 1, deafness, autosomal dominant 34 | Cryopyrin, nucleotide-binding oligomerization domain, leucine rich repeat and pyrin domain containing 3
Location: 1q44: 247332331 - 247449108 (+)
Gene type: protein_coding, 11 transcripts.
Scores: LoFtool: 0.003510 | pLI: 0.44726268 | LOEUF: 0.519
Normal function
The NLRP3 inflammasome is primarily expressed in myeloid cells and its importance to host defenses is reflected in its many complex levels of regulation, including by reactive oxygen species, calcium signaling, phosphorylation, ubiquitylation, prenylation, SUMOylation and microRNAs. The NLRP3 gene encodes the NACHT, LRR and PYD domains-containing protein 3 (NALP3), also known as cryopyrin. Cryopyrin is a nucleotide-binding domain and leucine-rich repeat containing (NLR) protein, cytosolic sensors that initiate and regulate the immune system's response to injury, toxins, or invasion by microorganisms. Cryopyrin recognizes bacterial particles, extracellular ATP, chemicals such as asbestos, silica, and uric acid crystals, and compounds released by injured cells. Once activated, cryopyrin and associated proteins assemble into inflammasomes, which are required to activate signaling cascades resulting in pro-inflammatory cytokine (i.e. IL-1beta, IL-18) production and pyroptosis.
Dysfunction and disease
Heterozygous NLRP3 GOF mutations are associated with various conditions grouped under the term Cryopyrinopathies (Cryopyrin-associated periodic syndrome or CAPS), or NLRP3-associated auto-inflammatory disease (NLRP3-AID). These conditions all present with cold-induced urticaria as a hallmark feature, but fall along a clinical continuum from milder FCAS (familial cold autoinflammatory syndrome) [OMIM:120100] to MWS (Muckle-Wells syndrome) [OMIM:191900] to severe NOMID (neonatal-onset multisystem inflammatory disease) or CINCA (chronic infantile neurological cutaneous and articular) syndrome [OMIM:607115] (PMID: 32546426). Patient cells spontaneously secrete high levels of IL-1beta, consistent with the spectacular therapeutic response that patients presenting with all three CAPS phenotypes have shown to IL-1 inhibition. However, CNS damage and SNHL may be irreversible despite therapy, especially if there is a long delay in treatment initiation after symptom onset. NOMID/CINCA is associated with systemic inflammation that is chronic and persistent, whereas flares in FCAS or MWS are triggered by cold temperature and humidity. FCAS is characterized by a delay in the onset of symptoms (minutes to hours) following cold exposure. Attacks are short-lived and involve fever, chills, arthritis/arthralgias, myalgias, headache, conjunctivitis, in addition to urticaria and swelling of the extremities. MWS is characterized by sensorineural hearing loss (SNHL) and the potential for SAA amyloidosis if untreated, in addition to attacks featuring ocular inflammation, fever, headache, and less commonly arthritis/arthralgias. NOMID/CINCA presents in the first days of life with TORCH-like craniofacial findings of frontal bossing and saddle-nose deformity along with manifestations of neurological (i.e. CNS lesions, chronic meningitis), cutaneous (diffuse non-pruritic urticarial rash), and articular (progressive deforming arthropathy, particularly of the knee) inflammation (PMID: 27927236, 31077002). The majority of disease-associated mutations are missense changes affecting exons 3 and 4, thus affecting the NACHT domain or nearby residues (PMID: 28191008). Those located around the ATP-binding pocket are usually inherited de novo and cause constitutive inflammasome activation, leading to the severe phenotypes of NOMID/CINCA, while others may destabilize interdomain interactions and lower the threshold for NLRP3 activation, leading to FCAS or MWS (PMID: 31189953). Several missense mutations in the autoinhibitory LRR domains have been identified in patients with late-onset symptoms, hearing loss and atypical presentations (PMID: 20131254, 15334500, 12032915) or even isolated SNHL [OMIM:617772] (PMID: 28847925). Moreover, recurrent low penetrance missense variants in multiple domains have also been identified, with patients showing more fever and gastrointestinal symptoms than “typical” CAPS patients and intermediate cellular phenotypes between wild-type and highly penetrant NLRP3 mutations on functional inflammasome studies (PMID: 28692792). Thus far, only one CAPS-associated mutation has been identified in the pyrin domain (PYD) of NLRP3 - specifically, a missense change in a MWS patient that increased its interaction with the PYD of ASC (apoptosis-associated speck-like protein) (PMID: 28229991). Of note, another rare exon 1 variant (Asp21His) affecting the PYD has been identified in Finnish families diagnosed with keratoendotheliitis fugax hereditaria, a cond [Load More]
[Reviewed by Xiao P. Peng on 2022-06-21 16:21:52]
Associated conditions
Please mind that full curation (inclusion of all published patients) of this gene has not started yet. Please contact us if you want to volunteer.
Transcripts of NLRP3
Name | ENSEMBL_ID | LRG_ID | CCDS_ID | MANE | Transcript.type | Exons | Canonical | CDS_length | REFSEQ_ID |
---|---|---|---|---|---|---|---|---|---|
202 | ENST00000348069.7 | protein_coding | 7 | No | 3824 | NM_183395 | |||
201 | ENST00000336119.8 | Select | protein_coding | 10 | Yes | 4187 | NM_001079821,NM_001243133,NM_004895 | ||
203 | ENST00000366496.7 | protein_coding | 8 | No | 3995 | NM_001127461 | |||
205 | ENST00000391827.3 | protein_coding | 9 | No | 4242 | NM_001127462 | |||
210 | ENST00000697350.1 | protein_coding | 10 | No | XM_047443534 |
Published variants
Found 532 variants
Please mind that full curation (inclusion of all published variants) of this gene has not started yet. Please contact us if you want to volunteer.