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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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TBX1
T-box transcription factor 1
Chromosome 22 Β· 22q11.21
NCBI Gene: 6899Ensembl: ENSG00000184058.16HGNC: HGNC:11592UniProt: A0A3B3IS18
85PubMed Papers
24Diseases
0Drugs
60Pathogenic Variants
FUNCTIONAL ROLE
Transcription Factor
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
sequence-specific double-stranded DNA bindingprotein bindingsoft palate developmentthymus development22q11.2 deletion syndromeconotruncal heart malformationsTetralogy of Fallotprostate carcinoma
✦AI Summary

TBX1 is a T-box transcription factor encoded on chromosome 22.2 that serves as a master regulator of embryonic development, particularly cardiovascular and craniofacial systems 1. The gene functions through epigenetic modifications rather than as a strong transcriptional activator or repressor, regulating diverse developmental processes 1. TBX1 is haploinsufficient, meaning loss of a single copy can cause disease 1. Mechanistically, TBX1 promotes pharyngeal arch segmentation and cardiac morphogenesis, working upstream of thymus and parathyroid gland development from the third pharyngeal pouch. After myocardial infarction, TBX1 in lymphatic endothelial cells promotes an immunosuppressive microenvironment through chemokine Ccl21 and integrin Icam1, facilitating cardiac repair 2. Clinically, TBX1 loss-of-function mutations cause 22q11.2 deletion syndrome (DiGeorge/velocardiofacial syndrome), the most common microdeletion syndrome affecting ~1:2000 individuals 34. This syndrome presents with conotruncal heart malformations including tetralogy of Fallot and ventricular septal defects 56, palatal defects, thymic hypoplasia causing immunodeficiency, hypocalcemia from parathyroid dysfunction, and neurodevelopmental abnormalities 6. Conversely, 22q11.2 duplication syndrome causes variable phenotypes including congenital heart disease, gastrointestinal complications, and developmental delays 7. The phenotypic severity correlates with the chr22 region affected, with TBX1 loss being the primary driver of cardiac and craniofacial manifestations 3.

Sources cited
1
TBX1 is a T-box transcription factor that is haploinsufficient, regulates genes through epigenetic modifications, and is neither a strong activator nor repressor
PMID: 28057265
2
TBX1 in lymphatic endothelial cells promotes immunosuppressive microenvironment after myocardial infarction through Ccl21 and Icam1, facilitating cardiac repair
PMID: 37625409
3
TBX1 loss is the most important cause of 22q11.2 deletion syndrome phenotype; this is one of the most common microdeletion syndromes
PMID: 25123976
4
Velo-cardio-facial syndrome (22q11.2 deletion) is one of the most common genetic syndromes affecting ~1:2000 individuals and most common syndrome of conotruncal heart malformations
PMID: 16282778
5
TBX1 mutations are associated with DiGeorge syndrome and conotruncal heart defects including ventricular septal defects
PMID: 38884729
6
22q11.2 deletion syndrome with TBX1 loss causes cardiovascular defects (tetralogy of Fallot, truncus arteriosus), palatal defects, hypocalcemia, thymic hypoplasia, and immunodeficiencies
PMID: 40038168
7
22q11.2 duplication syndrome affects ~1:700 of individuals with intellectual disability and presents with congenital heart disease, gastrointestinal complications, and developmental delays
PMID: 34845825
Disease Associationsβ“˜24
22q11.2 deletion syndromeOpen Targets
0.84Strong
conotruncal heart malformationsOpen Targets
0.67Moderate
Tetralogy of FallotOpen Targets
0.60Moderate
prostate carcinomaOpen Targets
0.52Moderate
Shprintzen-Goldberg syndromeOpen Targets
0.50Moderate
Nasal Cavity PolypOpen Targets
0.46Moderate
neurodegenerative diseaseOpen Targets
0.41Moderate
genetic disorderOpen Targets
0.37Weak
T-B- severe combined immunodeficiencyOpen Targets
0.37Weak
T-B+ severe combined immunodeficiencyOpen Targets
0.37Weak
T+ B+ severe combined immunodeficiencyOpen Targets
0.37Weak
hypertrophic cardiomyopathyOpen Targets
0.35Weak
KBG syndromeOpen Targets
0.34Weak
non-suppurative otitis mediaOpen Targets
0.34Weak
middle ear disorderOpen Targets
0.34Weak
adolescent idiopathic scoliosisOpen Targets
0.34Weak
suppurative otitis mediaOpen Targets
0.34Weak
Abnormal mastoid morphologyOpen Targets
0.34Weak
chronic rhinosinusitis with nasal polypsOpen Targets
0.34Weak
sinusitisOpen Targets
0.31Weak
Conotruncal heart malformationsUniProt
DiGeorge syndromeUniProt
Tetralogy of FallotUniProt
Velocardiofacial syndromeUniProt
Pathogenic Variants60
NM_001379200.1(TBX1):c.61G>A (p.Ala21Thr)Likely pathogenic
Velocardiofacial syndrome;DiGeorge syndrome|DiGeorge syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 21
NM_001379200.1(TBX1):c.1222G>T (p.Glu408Ter)Pathogenic
TBX1-related disorder|DiGeorge syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 408
NM_001379200.1(TBX1):c.711+1G>APathogenic
DiGeorge syndrome|TBX1-related disorder
β˜…β˜…β˜†β˜†2025
NM_001379200.1(TBX1):c.89_284del (p.Leu30fs)Pathogenic
DiGeorge syndrome|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 30
NM_001379200.1(TBX1):c.1063C>T (p.Gln355Ter)Pathogenic
DiGeorge syndrome
β˜…β˜…β˜†β˜†2024β†’ Residue 355
NM_001379200.1(TBX1):c.1057G>T (p.Glu353Ter)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2026β†’ Residue 353
NM_001379200.1(TBX1):c.1036+1G>TPathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2025
NM_001379200.1(TBX1):c.1122_1144del (p.Val377fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2025β†’ Residue 377
NM_001379200.1(TBX1):c.935+1G>ALikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001379200.1(TBX1):c.201dup (p.Gly68fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2025β†’ Residue 68
NM_001379200.1(TBX1):c.712-1G>TLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001379200.1(TBX1):c.1215dup (p.Asn406fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2025β†’ Residue 406
NM_001379200.1(TBX1):c.652C>T (p.Gln218Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 218
NM_001379200.1(TBX1):c.1036+2T>CPathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2024
NM_080646.2(TBX1):c.1072_1073del (p.Glu358fs)Likely pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2024β†’ Residue 358
NM_001379200.1(TBX1):c.364C>T (p.Gln122Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 122
NM_001379200.1(TBX1):c.1027del (p.Thr343fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 343
NM_001379200.1(TBX1):c.1015C>T (p.Gln339Ter)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 339
NM_001379200.1(TBX1):c.1206_1207insGAACCCCGAGC (p.Ser403fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 403
NM_001379200.1(TBX1):c.198_229dup (p.His77fs)Pathogenic
DiGeorge syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 77
View on ClinVar β†—
Related Genes
GP1BBProtein interaction89%SEPTIN5Protein interaction89%GNB1LProtein interaction81%TMEM26Protein interaction79%GSC2Protein interaction75%COMTProtein interaction75%
Tissue Expression6 tissues
Lung
100%
Ovary
32%
Liver
9%
Brain
7%
Heart
3%
Bone Marrow
0%
Gene Interaction Network
Click a node to explore
TBX1GP1BBSEPTIN5GNB1LTMEM26GSC2COMT
PROTEIN STRUCTURE
Preparing viewer…
PDB4A04 Β· 2.58 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.70LoF Tolerant
pLIβ“˜
0.12Tolerant
Observed/Expected LoF0.44 [0.29–0.70]
RankingsWhere TBX1 stands among ~20K protein-coding genes
  • #5,642of 20,598
    Most Researched85
  • #1,168of 5,498
    Most Pathogenic Variants60 Β· top quartile
  • #5,359of 17,882
    Most Constrained (LOEUF)0.70
Genes detectedTBX1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
22q11.2 duplications: Expanding the clinical presentation.
PMID: 34845825
Am J Med Genet A Β· 2022
1.00
2
PMID: 20301696
0.90
3
PMID: 25905388
0.80
4
Central 22q11.2 deletions.
PMID: 25123976
Am J Med Genet A Β· 2014
0.70
5
Human Genetics of Ventricular Septal Defect.
PMID: 38884729
Adv Exp Med Biol Β· 2024
0.60