SMO

Chr 7AR

smoothened, frizzled class receptor

Also known as: CRJS, FZD11, Gx, PHLS, SMOH

The protein is a G protein-coupled receptor that transduces hedgehog signaling by interacting with the patched protein receptor complex. Gain-of-function mutations cause Curry-Jones syndrome and Pallister-Hall-like syndrome through autosomal recessive inheritance. The pathogenic mechanism involves disrupted hedgehog pathway signaling leading to developmental abnormalities.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
MultiplemechanismARLOEUF 0.683 OMIM phenotypes
Clinical SummarySMO
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Gene-Disease Validity (ClinGen)
congenital hypothalamic hamartoma syndrome · ARModerate

Moderate evidence — consider for supplementary testing

2 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
32 unique Pathogenic / Likely Pathogenic· 58 VUS of 161 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.68LOEUF
pLI 0.000
Z-score 3.08
OE 0.46 (0.310.68)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
1.90Z-score
OE missense 0.75 (0.690.82)
345 obs / 459.4 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.46 (0.310.68)
00.351.4
Missense OE0.75 (0.690.82)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 17 / 37.3Missense obs/exp: 345 / 459.4Syn Z: 0.10
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveSMO-related Curry-Jones syndromeGOFAD
strongSMO-related developmental disorderLOFAR
DN
0.6646th %ile
GOF
0.77top 25%
LOF
0.3260th %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

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

GOFThe identification of a gain-of-function mutation in SMO causing a type 1 mosaic form of BCNS further expands our understanding of the pathogenesis of BCC, with implications for the treatment of these tumours, whether sporadic or inherited.PMID:26822128

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

ClinVar Variant Classifications

161 submitted variants in ClinVar

Classification Summary

Pathogenic30
Likely Pathogenic2
VUS58
Likely Benign39
Benign26
Conflicting3
30
Pathogenic
2
Likely Pathogenic
58
VUS
39
Likely Benign
26
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
2
7
21
0
30
Likely Pathogenic
0
2
0
0
2
VUS
2
49
4
3
58
Likely Benign
0
5
20
14
39
Benign
0
4
17
5
26
Conflicting
3
Total4676222158

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

View in ClinVar →

Protein Context — Lollipop Plot

SMO · 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

Breast CancerCholangiocarcinomaColorectal Cancer

Basket Study of Entrectinib (RXDX-101) for the Treatment of Patients With Solid Tumors Harboring NTRK 1/2/3 (Trk A/B/C), ROS1, or ALK Gene Rearrangements (Fusions)

ACTIVE NOT RECRUITING
NCT02568267Phase PHASE2Hoffmann-La RocheStarted 2015-11-19
Entrectinib
Non-Small Cell Lung Cancer

A Study of Osimertinib With or Without Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor Non-Small Cell Lung Cancer (FLAURA2)

ACTIVE NOT RECRUITING
NCT04035486Phase PHASE3AstraZenecaStarted 2019-07-02
OsimertinibPemetrexed/CarboplatinPemetrexed/Cisplatin
Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR)

RECRUITING
NCT03297606Phase PHASE2Canadian Cancer Trials GroupStarted 2018-03-23
OlaparibDasatinibNivolumab plus Ipilimumab
Solid TumorNon-Small-Cell Lung CancerPancreatic Ductal Adenocarcinoma

A Study to Find a Suitable Dose of ASP5834 in Adults With Solid Tumors

RECRUITING
NCT07094204Phase PHASE1Astellas Pharma IncStarted 2025-07-25
ASP5834panitumumab
Advanced Soft-tissue Sarcoma

LINNOVATE: Lurbinectedin, Ipilimumab and Nivolumab for Soft Tissue Sarcoma

RECRUITING
NCT05876715Phase PHASE1, PHASE2ERLINDA M GORDONStarted 2023-06-07
Lurbinectedin
Advanced Solid Tumor

Study of CX-5461 in Patients With Solid Tumours and BRCA1/2, PALB2 or Homologous Recombination Deficiency (HRD) Mutation

RECRUITING
NCT04890613Phase PHASE1Senhwa Biosciences, Inc.Started 2021-09-08
CX-5461
Neoplasms

Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T Cells

RECRUITING
NCT03435796Phase PHASE2, PHASE3CelgeneStarted 2018-07-19
Gene-modified (GM) T cell therapy
Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen

Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen

RECRUITING
NCT06127407Phase PHASE3Servier Bio-Innovation LLCStarted 2024-07-09
Ivosidenib 500mgPlacebo
Advanced Non-small Cell Lung CancerHER2 Mutation

A Study to Learn More About How Well Sevabertinib Works and How Safe it is Compared With Standard Treatment, in Participants Who Have Advanced Non-small Cell Lung Cancer (NSCLC) With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)

RECRUITING
NCT06452277Phase PHASE3BayerStarted 2024-08-28
SevabertinibPembrolizumabCisplatin
Degenerative SpondylolisthesisLumbar Spinal Stenosis

LimiFlex Clinical Trial for the Treatment of Degenerative Spondylolisthesis With Spinal Stenosis

ACTIVE NOT RECRUITING
NCT03115983Phase NAEmpirical Spine, Inc.Started 2017-07-17
LimiFlexFusion InstrumentationDecompression
Hereditary Angioedema

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

ACTIVE NOT RECRUITING
NCT06634420Phase PHASE3Intellia TherapeuticsStarted 2025-01-15
NTLA-2002Normal Saline IV Administration
Intracranial MeningiomaRecurrent MeningiomaNF2 Gene Mutation

Vismodegib, FAK Inhibitor GSK2256098, Capivasertib, and Abemaciclib in Treating Patients With Progressive Meningiomas

RECRUITING
NCT02523014Phase PHASE2Alliance for Clinical Trials in OncologyStarted 2015-09-28
VismodegibFAK Inhibitor GSK2256098Capivasertib
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
[Medulloblastoma].
Kanemura Y·No Shinkei Geka
2022
[Meningioma].
Sasaki H·No Shinkei Geka
2023
Simpson-Golabi-Behmel syndrome.
Vaisfeld A et al.·Am J Med Genet C Semin Med Genet
2024Review
Top 5 results · since 2015Search PubMed ↗