TWIST1

Chr 7AD

twist family bHLH transcription factor 1

Also known as: ACS3, BPES2, BPES3, CRS, CRS1, CSO, SCS, SWCOS

The protein is a basic helix-loop-helix transcription factor that binds to DNA E box sequences and regulates transcription of genes controlling cranial suture closure, neural tube closure, and limb development. Mutations cause autosomal dominant craniosynostosis syndromes including Saethre-Chotzen syndrome, Robinow-Sorauf syndrome, and Sweeney-Cox syndrome, typically presenting with premature skull suture fusion, ptosis, and hypertelorism. The pathogenic mechanism involves haploinsufficiency leading to disrupted transcriptional regulation of developmental genes.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismADLOEUF 1.064 OMIM phenotypes
Clinical SummaryTWIST1
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Gene-Disease Validity (ClinGen)
Sweeney-Cox syndrome · ADLimited

Limited evidence — not for standalone diagnostic reporting

3 total gene-disease associations curated

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.22) despite low pLI — interpret in context.
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Clinical Trials
8 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — TWIST1
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.06LOEUF
pLI 0.345
Z-score 1.52
OE 0.22 (0.081.06)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
1.06Z-score
OE missense 0.67 (0.530.84)
53 obs / 79.5 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.22 (0.081.06)
00.351.4
Missense OE0.67 (0.530.84)
00.61.4
Synonymous OE1.60
01.21.6
LoF obs/exp: 1 / 4.5Missense obs/exp: 53 / 79.5Syn Z: -2.88
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveTWIST1-related Saethre-Chotzen syndromeLOFAD
DN
0.5575th %ile
GOF
0.3491th %ile
LOF
0.63top 25%

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

LOF1 literature citation
DN1 literature citation
GOF1 literature citation

Literature Evidence

DNPhosphorylation-dead TWIST1 acts as dominant negative and fully prevents EMT and tumor formation in vivo, thereby highlighting the significance of LIMK2-TWIST1 signaling axis in CRPC.PMID:30716360
GOFWhile the etiology of nonsyndromic craniosynostosis remains to be deciphered, gain-of-function mutations in FGFR1-3 and TWIST1 were found to be responsible for more than 3/4 of the most commonly encountered craniofacial syndromes.PMID:30976284
LOFSaethre-Chotzen syndrome caused by TWIST 1 gene mutations: functional differentiation from Muenke coronal synostosis syndromePMID:16251895

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

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

TWIST1 · 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

Craniosynostosis

Network Of Clinical Research Studies On Craniosynostosis, Skull Malformations With Premature Fusion Of Skull Bones

ACTIVE NOT RECRUITING
NCT03025763Icahn School of Medicine at Mount SinaiStarted 2015-01-13
Craniosynostosis Network Environmental Survey2D/3D PhotographyBuccal Swab Cell Sampling
CraniosynostosesCrouzon SyndromeSaethre Chotzen Syndrome

ASO Treatment for Syndromic Craniosynostoses

NOT YET RECRUITING
NCT07535372Fondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2026-04-20
Design of patient specific ASO
Myelodysplastic SyndromesAcute Myelogenous Leukemia

Role of BMP Pathway in MDS Progression

NOT YET RECRUITING
NCT06175923Hospices Civils de LyonStarted 2024-01-27
Collection of EDTA (disodium salt of ethylenediaminetetraacetic acid) tubes of marrow during routine care
Hypophosphatasia

The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia

RECRUITING
NCT07390240AstraZenecaStarted 2025-12-29
Prader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome

GROWing Up With Rare GENEtic Syndromes

RECRUITING
NCT04463316dr. Laura C. G. de Graaff-HerderStarted 2018-10-01
Retrospective file studies
AortopathiesThoracic Aortic AneurysmAortic Valve Disease

Pathogenetic Basis of Aortopathy and Aortic Valve Disease

ACTIVE NOT RECRUITING
NCT03440697Yale UniversityStarted 2015-12-10
Craniofacial Defects, SubtleSkull DefectCraniosynostoses

Functionalized Bioink Delivering Biomolecules for the Treatment of Craniofacial Diseases

ACTIVE NOT RECRUITING
NCT06533150Fondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-10-07
multiomic profiling
AIDS-Related Kaposi SarcomaSkin Kaposi Sarcoma

Nirogacestat in Patients With Kaposi Sarcoma

NOT YET RECRUITING
NCT07539454Phase PHASE2AIDS Malignancy ConsortiumStarted 2026-09-17
Biospecimen CollectionChest RadiographyComputed Tomography
Clinical Literature
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