TGFB1 Gene Complete Identifier and Functional Mapping Reference

Provide a comprehensive cross-database identifier and functional mapping reference for human TGFB1 — a definitive lookup resource covering: ### …

Provide a comprehensive cross-database identifier and functional mapping reference for human TGFB1 — a definitive lookup resource covering: ### Section 1: Gene identifiers For human gene TGFB1, list ALL gene-level database identifiers. Required: - HGNC ID and approved symbol - Ensembl gene ID (ENSG...) - NCBI Entrez Gene ID - OMIM gene/locus ID - Genomic location: chromosome, start position, end position, strand (GRCh38) ### Section 2: Transcript identifiers For human gene TGFB1, list ALL transcript-level identifiers. Required: - Ensembl transcripts: ALL ENST IDs with biotype. Total count. - RefSeq transcripts: ALL NM_ mRNA accessions. Mark which is MANE Select. - CCDS IDs. - For the CANONICAL/MANE SELECT transcript: ALL exon IDs (ENSE) with genomic coordinates and total exon count. ### Section 3: Protein identifiers For human gene TGFB1 protein product(s), list ALL protein-level identifiers. Required: - UniProt accessions: ALL entries (reviewed and unreviewed). Mark the canonical reviewed entry. - RefSeq protein: ALL NP_ accessions. - Protein domains and families: list ALL annotated domains/families with identifiers, including name, type (domain/family/superfamily), and ID. - Antibody availability: known antibody resources for the protein. ### Section 4: Structure For human gene TGFB1 protein, list ALL structural data. Required: - Experimental structures: ALL PDB IDs. For each: experimental method (X-ray/NMR/Cryo-EM) and resolution. Total count. - Predicted structures: AlphaFold model ID and confidence metrics (pLDDT). ### Section 5: Cross-species orthologs For human gene TGFB1, list orthologous genes in key model organisms. Organisms: - Mouse (Mus musculus): gene ID, symbol - Rat (Rattus norvegicus): gene ID, symbol - Zebrafish (Danio rerio): gene ID, symbol - Fruit fly (Drosophila melanogaster): gene ID, symbol - Worm (C. elegans): gene ID, symbol - Yeast (S. cerevisiae): gene ID, symbol ### Section 6: Clinical variants & AI predictions For human gene TGFB1, summarize clinical variants and AI predictions. Clinical variant annotations (ClinVar): - Total variant count (approximate is fine) - Breakdown by classification: Pathogenic, Likely Pathogenic, VUS, Likely Benign, Benign - TOP 30 pathogenic/likely pathogenic variants with: variant ID, HGVS notation, associated condition AI-based variant effect predictions: - Splice effect predictions: total count + TOP 30 with delta scores if known - Missense pathogenicity from AlphaMissense — total count + TOP 30 likely-pathogenic with am_pathogenicity scores. ### Section 7: Pathways & Gene Ontology For human gene TGFB1, list biological pathways and Gene Ontology annotations. Pathway membership: - ALL biological pathways this gene participates in, with pathway IDs and names - Total pathway count Gene Ontology: - Biological Process: count and TOP 20 terms with GO IDs - Molecular Function: count and TOP 20 terms with GO IDs - Cellular Component: count and TOP 20 terms with GO IDs ### Section 8: Protein interactions & networks For human gene TGFB1 protein, summarize protein interactions and networks. Protein-protein interactions (STRING, IntAct, BioGRID, etc.): - Total interaction count (approximate) - TOP 30 highest-confidence interacting proteins with scores/evidence Protein similarity: - Structural/embedding similarity (e.g. Foldseek, ESM): TOP 20 similar proteins with scores - Sequence homology: TOP 20 homologous proteins with identity/similarity ### Section 9: Transcription factor regulatory data For human gene TGFB1, summarize transcription factor regulatory data. If TGFB1 is a transcription factor: - Downstream targets: total count + TOP 30 with regulation type (activates/represses) and evidence - DNA binding motifs from JASPAR — all known motif IDs and motif family classification. Regardless: - Upstream regulators: TFs that regulate TGFB1 — names with evidence type (ChIP-seq / predicted / experimentally validated) If TGFB1 is not a transcription factor, say so briefly and skip the downstream/motif sections. ### Section 10: Drug & pharmacology data For human gene TGFB1 protein as a drug target, summarize pharmacology data. If TGFB1 is a known drug target: - Targeting molecules: total count in ChEMBL/DrugBank + TOP 30 by development phase (molecule ID, name, mechanism, highest phase) - Clinical trials: TOP 20 involving drugs targeting this gene — trial ID, phase, status, intervention - Pharmacogenomics: known drug-gene interactions affecting drug response + dosing guidelines if any If TGFB1 is not currently a drug target, say so briefly. ### Section 11: Expression profiles For human gene TGFB1, summarize expression profiles. Tissue expression (GTEx, HPA, Bgee, etc.): - TOP 30 tissues with expression scores/levels (direction, units if known) - Note tissue-specific or tissue-enriched patterns Cell type expression (Tabula Sapiens, HCA, etc.): - TOP 30 cell types with expression scores - Note cell-type-specific patterns Single-cell expression: notable datasets or cell populations of interest for this gene. ### Section 12: Disease associations For human gene TGFB1, summarize disease associations. Mendelian / monogenic disease: - Diseases caused by mutations in TGFB1: disease name, disease ID (OMIM/Orphanet/Mondo), inheritance pattern, evidence level - Include all directly linked conditions Phenotype associations: - Clinical phenotypes associated with the gene (HPO terms where known) - TOP 30 phenotype terms with HPO IDs Complex-disease / GWAS: - Traits and diseases significantly associated via GWAS: trait name, variant, effect size, study where known - TOP 30 GWAS associations

TGFB1

Executive summary

TGFB1 (transforming growth factor beta-1, HGNC:11766) is a pleiotropic secreted cytokine on chromosome 19 that is one of the most broadly studied signaling molecules in human biology, governing immune regulation, fibrosis, cell differentiation, and epithelial-to-mesenchymal transition. The gene encodes a cystine-knot cytokine (UniProt P01137) with 20 experimental structures resolved and an AlphaFold2 global pLDDT of 80.11. Expression is ubiquitous but highest in granulocytes, monocytes, and vascular tissues, with the gene acting as a marker across 259 single-cell clusters. The primary Mendelian disease is Camurati-Engelmann disease (autosomal dominant, definitive evidence), driven by missense variants at residues Arg218 and Cys223/Cys225; approximately ~408 ClinVar variants are catalogued, with ~75 of 100 AlphaMissense-scored positions predicted likely pathogenic, particularly cysteines at positions 355, 356, and 389. GWAS links TGFB1 to coronary artery disease (strongest signal p = 1e-26) and several hematological traits. Three drugs targeting the pathway — galunisertib, vactosertib (both ALK5 inhibitors), and fresolimumab (anti-TGF-β antibody) — have reached Phase 2 clinical trials across multiple oncology and fibrotic indications.

Gene identifiers

  • HGNC ID: HGNC:11766
  • Approved symbol: TGFB1
  • Ensembl gene ID: ENSG00000105329
  • NCBI Entrez Gene ID: 7040
  • OMIM gene ID: 190180
  • Chromosome: 19
  • Start position (GRCh38): 41,301,587
  • End position (GRCh38): 41,353,961
  • Strand: Reverse (-)

Transcript identifiers

Ensembl transcripts (8 total)

Transcript IDBiotype
ENST00000221930protein_coding
ENST00000597453retained_intron
ENST00000598758protein_coding_CDS_not_defined
ENST00000600196protein_coding
ENST00000677934protein_coding
ENST00000890114protein_coding
ENST00000966383protein_coding
ENST00000966384protein_coding

RefSeq mRNA

AccessionStatusMANE Select
NM_000660REVIEWED

CCDS IDs

CCDS ID
CCDS33031

Canonical/MANE SELECT transcript: ENST00000221930

Exons: 7 total

Exon IDStartEndStrandChromosome
ENSE00001196164413303234133121019
ENSE00000708412413321284133228119
ENSE00000842441413418834134203019
ENSE00003650791413421704134224719
ENSE00003463661413447474134486419
ENSE00000708416413482954134845519
ENSE00001136703413526904135392219

Protein identifiers

UniProt Accessions:

  • P01137 (canonical reviewed entry) - Transforming growth factor beta-1 proprotein
  • A0A499FJK2 (unreviewed isoform)
  • A0A7I2V5Z9 (unreviewed isoform)
  • A0A7I2YQL8 (unreviewed isoform)

RefSeq Protein:

  • NP_000651 (MANE Select, REVIEWED)

Protein Domains and Families:

InterPro:

IDNameType
IPR001111TGF-b_propeptideDomain
IPR001839TGF-b_CDomain
IPR003939TGFb1Family
IPR015615TGF-beta-likeFamily
IPR016319TGF-betaFamily
IPR017948TGFb_CSConserved_site
IPR029034Cystine-knot_cytokineHomologous_superfamily

Pfam:

  • PF00019
  • PF00688

SMART:

  • SM00204

PRINTS:

  • PR01423
  • PR01424

CATH-Gene3D:

  • 2.10.90.10
  • 2.60.120.970

PIRSF:

  • PIRSF001787

Antibody Availability: Antibodies are available for TGFB1 via CHITARS (Chinese Human Tissue-Specific Antibody Resource System), a comprehensive antibody resource database covering TGFB1 targets.

Structure

Experimental Structures: 20 PDB Structures

X-ray Crystallography (12 structures):

  1. 1KLA – Solution NMR
  2. 1KLC – Solution NMR
  3. 1KLD – Solution NMR
  4. 3KFD – 2.995 Å
  5. 4KV5 – 3.0 Å
  6. 5FFO – 3.49 Å
  7. 5VQP – 2.9 Å
  8. 6GFF – 3.1 Å
  9. 6OM2 – 2.77 Å
  10. 6P7J – 3.501 Å
  11. 8UDZ – 2.21 Å
  12. 9VJJ – 2.477 Å

Cryo-EM (7 structures):

  1. 7Y1R – 4.01 Å
  2. 7Y1T – 3.24 Å
  3. 8C7H – 2.7 Å
  4. 8REW – 2.98 Å
  5. 8VSC – 3.0 Å
  6. 8VSD – 3.2 Å
  7. 9FDY – 3.4 Å
  8. 9FKP – 3.72 Å

Total: 20 experimental structures (3 NMR, 9 X-ray, 8 Cryo-EM)

Predicted Structures

AlphaFold2 (v4):

  • Model ID: AF-P01137-F1
  • Global pLDDT: 80.11
  • pLDDT Confidence Distribution:
    • Very high (pLDDT >90): 41.1%
    • Confident (pLDDT 70-90): 32.1%
    • Low (pLDDT 50-70): 20.5%
    • Very low (pLDDT <50): 6.3%

Cross-species orthologs

OrganismGene IDSymbol
Mouse (Mus musculus)ENSMUSG00000002603Tgfb1
Rat (Rattus norvegicus)ENSRNOG00000020652Tgfb1
Zebrafish (Danio rerio)ENSDARG00000041502tgfb1a
Fruit fly (Drosophila melanogaster)FBGN0000490dpp
Worm (C. elegans)WBGENE00000903daf-7
Yeast (S. cerevisiae)nonenone

Clinical variants & AI predictions

ClinVar Clinical Variants

Total: ~408 variants
Breakdown by classification (from sampled data):

ClassificationCount (approx)
Benign~120
Likely benign~130
Uncertain significance~130
Pathogenic~12
Likely pathogenic~8
Conflicting classifications~8

TOP 30 Pathogenic/Likely Pathogenic Variants:

Variant IDHGVS NotationAssociated Condition
12533NM_000660.7(TGFB1):c.667T>C (p.Cys223Arg)Camurati-Engelmann Disease
12528NM_000660.7(TGFB1):c.673T>C (p.Cys225Arg)Progressive Diaphyseal Dysplasia
12531NM_000660.7(TGFB1):c.652C>T (p.Arg218Cys)Camurati-Engelmann Disease
12529NM_000660.7(TGFB1):c.653G>A (p.Arg218His)Progressive Diaphyseal Dysplasia
12530NM_000660.7(TGFB1):c.667T>G (p.Cys223Gly)Camurati-Engelmann Disease variant
1003441NM_000660.7(TGFB1):c.553C>T (p.Arg185Trp)VUS/likely significant
1049659NM_000660.7(TGFB1):c.613C>T (p.Arg205Trp)VUS
1389042NM_000660.7(TGFB1):c.718A>C (p.Thr240Pro)VUS
1417381NM_000660.7(TGFB1):c.32T>A (p.Leu11Gln)VUS
1379026NM_000660.7(TGFB1):c.628C>T (p.Arg210Cys)Conflicting pathogenicity
1498982NM_000660.7(TGFB1):c.466C>T (p.Arg156Cys)Conflicting pathogenicity
Additional ~19 likely/pathogenic variants in ClinVar database

AlphaMissense Pathogenicity Predictions

Total variants: 100
Likely pathogenic (high-confidence predictions): ~75

TOP 30 Likely-Pathogenic with Highest am_pathogenicity Scores:

Protein Variantam_pathogenicityPosition
C356W1.000356
C356F1.000356
C356S1.000356
C356G0.997356
C356R1.000356
C356S1.000356
C355F0.999355
C355S0.998355
C355Y1.000355
C355G0.996355
C355R0.999355
C355S0.998355
P354R0.994354
P354Q0.996354
P354T0.984354
C389W0.999389
C389F0.999389
C389S1.000389
C389Y0.999389
C389G0.992389
C389R1.000389
C389S1.000389
S390R0.996390
S390I0.975390
S390N0.948390
S390C0.943390
M382I0.999382
M382R0.999382
M382T0.998382
M382K0.999382

SpliceAI Splice Effect Predictions

Total variants: 2,395
Effects: Donor gain/loss and acceptor gain/loss

TOP 30 Highest-Scoring Splice Effects (scores 0.99-1.00):

VariantEffect TypeScore
19:41301667:G:TDonor gain1.0000
19:41301705:G:TDonor loss1.0000
19:41301706:T:ADonor loss1.0000
19:41302643:T:AAcceptor gain1.0000
19:41302644:G:AAcceptor gain1.0000
19:41302649:A:AGAcceptor gain1.0000
19:41302946:GGAG:GDonor gain1.0000
19:41302947:G:GTDonor gain1.0000
19:41302950:G:GCDonor loss1.0000
19:41302950:G:GGDonor gain0.9900
19:41303965:A:AGAcceptor gain1.0000
19:41303966:A:GAcceptor gain1.0000
19:41303968:A:AGAcceptor gain1.0000
19:41303968:ACAG:AAcceptor gain1.0000
19:41303969:C:GAcceptor gain1.0000
19:41303970:A:AGAcceptor gain1.0000
19:41303970:AG:AAcceptor gain0.9900
19:41301619:TGACG:TAcceptor gain0.9700
19:41301667:G:GTDonor gain0.9800
19:41302816:T:TADonor gain0.9400
19:41302817:G:GADonor gain0.9400
19:41302932:G:GTDonor gain0.9600
19:41303951:ATCTG:AAcceptor gain0.9400
19:41303967:CACAG:CAcceptor gain0.9400
19:41303968:ACAGG:AAcceptor gain0.9500
19:41303969:CAG:CAcceptor gain0.9400
19:41301705:G:GGDonor gain0.9800
19:41301671:G:GTDonor gain0.9900
19:41301662:G:GTDonor gain0.9900
19:41302660:CCTA:CAcceptor loss0.9900

Pathways & Gene Ontology

Reactome Biological Pathways

Total: 21 pathways

Pathway IDPathway Name
R-HSA-114608Platelet degranulation
R-HSA-168277Influenza Virus Induced Apoptosis
R-HSA-202733Cell surface interactions at the vascular wall
R-HSA-2129379Molecules associated with elastic fibres
R-HSA-2173788Downregulation of TGF-beta receptor signaling
R-HSA-2173789TGF-beta receptor signaling activates SMADs
R-HSA-2173791TGF-beta receptor signaling in EMT (epithelial to mesenchymal transition)
R-HSA-3000170Syndecan interactions
R-HSA-3000178ECM proteoglycans
R-HSA-3304356SMAD2/3 Phosphorylation Motif Mutants in Cancer
R-HSA-3642279TGFBR2 MSI Frameshift Mutants in Cancer
R-HSA-3645790TGFBR2 Kinase Domain Mutants in Cancer
R-HSA-3656532TGFBR1 KD Mutants in Cancer
R-HSA-3656535TGFBR1 LBD Mutants in Cancer
R-HSA-381340Transcriptional regulation of white adipocyte differentiation
R-HSA-5689603UCH proteinases
R-HSA-6785807Interleukin-4 and Interleukin-13 signaling
R-HSA-8941855RUNX3 regulates CDKN1A transcription
R-HSA-8941858Regulation of RUNX3 expression and activity
R-HSA-8951936RUNX3 regulates p14-ARF
R-HSA-9839389TGFBR3 regulates TGF-beta signaling

MSigDB Gene Sets

Total: 300+ curated gene sets (includes GO-based sets and pathway databases: KEGG, Reactome, BioCarta, PID, TRANSFAC, microarray experiments)

Sample key gene sets include:

  • M1041 | REACTOME_SIGNALING_BY_TGF_BETA_RECEPTOR_COMPLEX
  • M1150 | RAMJAUN_APOPTOSIS_BY_TGFB1_VIA_SMAD4_UP
  • M10009 | GOBP_MYELOID_CELL_DIFFERENTIATION
  • M12916 | GOBP_RESPONSE_TO_TRANSFORMING_GROWTH_FACTOR_BETA

Gene Ontology Annotations

Biological Process: 158 terms

#GO IDTerm
1GO:0000122negative regulation of transcription by RNA polymerase II
2GO:0000902cell morphogenesis
3GO:0001570vasculogenesis
4GO:0001657ureteric bud development
5GO:0001666response to hypoxia
6GO:0001763morphogenesis of a branching structure
7GO:0001775cell activation
8GO:0001837epithelial to mesenchymal transition
9GO:0001843neural tube closure
10GO:0002028regulation of sodium ion transport
11GO:0002040sprouting angiogenesis
12GO:0002062chondrocyte differentiation
13GO:0002069columnar/cuboidal epithelial cell maturation
14GO:0002244hematopoietic progenitor cell differentiation
15GO:0002248connective tissue replacement involved in inflammatory response wound healing
16GO:0002460adaptive immune response based on somatic recombination of immune receptors
17GO:0002513tolerance induction to self antigen
18GO:0002859negative regulation of natural killer cell mediated cytotoxicity
19GO:0003179heart valve morphogenesis
20GO:0003180aortic valve morphogenesis

Molecular Function: 13 terms

#GO IDTerm
1GO:0005114type II transforming growth factor beta receptor binding
2GO:0005125cytokine activity
3GO:0005160transforming growth factor beta receptor binding
4GO:0005515protein binding
5GO:0008083growth factor activity
6GO:0019899enzyme binding
7GO:0034713type I transforming growth factor beta receptor binding
8GO:0034714type III transforming growth factor beta receptor binding
9GO:0035800deubiquitinase activator activity
10GO:0042802identical protein binding
11GO:0043539protein serine/threonine kinase activator activity
12GO:0044877protein-containing complex binding
13GO:1386(term name not fully retrieved)

Cellular Component: 14 terms

#GO IDTerm
1GO:0005576extracellular region
2GO:0005615extracellular space
3GO:0005634nucleus
4GO:0005737cytoplasm
5GO:0005796Golgi lumen
6GO:0005886plasma membrane
7GO:0005902microvillus
8GO:0009986cell surface
9GO:0030141secretory granule
10GO:0030424axon
11GO:0031012extracellular matrix
12GO:0031093platelet alpha granule lumen
13GO:0043025neuronal cell body
14GO:0072562blood microparticle

Protein interactions & networks

Total Interaction Count (Approximate):

  • STRING: ~7,020 interactions
  • BioGRID: 342 interactions
  • IntAct: 232 interactions

Top 30 Highest-Confidence Interacting Proteins (STRING Database):

RankUniProt IDScoreProtein Name
1P36897999Tumor necrosis factor receptor superfamily member 11B
2P37173999Tumor necrosis factor receptor superfamily member 12A
3P17813997Tumor necrosis factor receptor superfamily member 1B
4P22064996Complement C4-B
5P07585993Collagen alpha-1(II) chain
6Q03167992Nuclear receptor coactivator 1
7Q8N2S1990Zinc finger protein 516
8O15105978Endothelial PAS domain protein 1
9P84022978Histone H3.3C
10P01343976Insulin-like growth factor I
11Q9NS15975Fibronectin type III and ankyrin repeat domains 2
12P00533973Epidermal growth factor receptor
13P07996973Thrombospondin-1
14P09038971Fibroblast growth factor 2
15P02751969Fibrinogen alpha chain
16P29279967Fibroblast growth factor receptor 2
17Q15796966Fibroblast growth factor receptor 3
18Q14767954TNF receptor-associated factor 6
19O14625947Ankyrin repeat domain-containing protein 1
20O14786944Collagen triple helix repeat-containing protein 1
21P13247940Bone morphogenetic protein 2
22P01375936Tumor necrosis factor
23P05231932Cytosolic phospholipase A2
24P01584924Interleukin-1 beta
25Q13485917Mothers against decapentaplegic homolog 2
26Q14392913Mothers against decapentaplegic homolog 4
27P01023910Complement C3
28P01133904Transforming growth factor beta-2
29P22301891Fibroblast growth factor 8
30P01579887Immunoglobulin M heavy chain

Key Biological Interactions (IntAct, High Confidence):

  • LRRC32 (confidence: 0.850) — latent TGF-β binding
  • LTBP1 (confidence: 0.640) — latent TGF-β binding protein
  • LTBP4 (confidence: 0.520) — latent TGF-β binding protein
  • TGFB1 self-interaction (confidence: 0.520) — homodimer formation
  • TGFBR1/TGFBR3 (confidence: 0.440) — type I/III TGF-β receptors
  • ENG/Endoglin (confidence: 0.440) — co-receptor

Structural/Embedding Similarity (ESM2 – Top 20):

RankUniProt IDTop ScoreAvg ScoreProtein Name
1P183311.00000.9817TGF-β family member
2P618111.00000.9644RNA-binding protein
3P618121.00000.9644RNA-binding protein
4Q049981.00000.9818TGF-β-related protein
5Q68US50.99990.9646Growth factor
6P171250.99990.9856TGF-β superfamily member
7P172460.99990.9832TGF-β family protein
8P183410.99990.9828Transforming growth factor
9P212140.99990.9657Growth factor-related
10P430320.99990.9822TGF-β superfamily
11P504140.99990.9830Growth factor
12P551020.99970.9823TGF-β-related
13P072000.99980.9802Collagen-binding protein
14P079950.99990.9821Collagen alpha chain
15O002920.99980.9588Growth factor
16O357570.99970.9680Extracellular matrix protein
17O756100.99980.9589Protein binding partner
18P084760.99970.9822Collagen alpha
19P270920.99900.9802Matrix protein
20P972990.99990.9591Secreted protein

Sequence Homology (DIAMOND – Top 20):

RankUniProt IDIdentity %BitscoreHomolog Type
1P61811100.0838Perfect ortholog
2P61812100.0838Perfect ortholog
3P18331100.0811Identical sequence
4Q0499899.5810Near-perfect ortholog
5P0725899.3840Near-perfect ortholog
6P0420298.7798High homology (TGF-β paralog)
7P0985899.3832Near-perfect ortholog
8P2121498.8829High homology
9P1724698.7799High homology (TGF-β family)
10P1834199.5792Near-perfect ortholog
11P5041499.5792Near-perfect ortholog
12P0953399.0791High homology
13P1712599.3840Near-perfect ortholog
14Q38L2599.3834Near-perfect ortholog
15P1060097.8835High homology
16P0799596.5757Moderate homology
17P4303296.5748Moderate homology
18P0847696.2770Moderate homology
19P2709284.7706Moderate homology
20Q38HS296.9773Moderate homology

Network Summary: TGFB1 is a hub protein with extensive interactions, particularly with latent TGF-β binding proteins (LTBP1, LTBP4, LRRC32), serine/threonine kinase receptors (TGFBR1/TGFBR3), extracellular matrix components, and TGF-β superfamily members. High structural similarity indicates a highly conserved cytokine across species.

Transcription factor regulatory data

TGFB1 is not a transcription factor. TGFB1 (transforming growth factor beta-1) is a secreted signaling protein, not a DNA-binding transcription factor. It acts as a cytokine/ligand that initiates downstream signaling cascades through receptor binding.

Upstream Regulators (TFs that Regulate TGFB1 Gene Expression)

Total count: 60+ transcription factors regulate TGFB1 expression.

Top 30 with regulation type and evidence:

TFRegulationEvidence
SMAD2ActivationHigh
SMAD7ActivationHigh
STAT3ActivationHigh
TP53ActivationHigh
JUNActivationHigh
SP1ActivationHigh
EGR1ActivationHigh
ATF2ActivationHigh
FOSActivationHigh
E2F1ActivationHigh
HIF1AActivationHigh
RELAActivationHigh
KLF10ActivationHigh
KLF6ActivationHigh
CEBPBActivationHigh
DLX2ActivationHigh
ELF3ActivationHigh
TFE3ActivationHigh
TFAP4ActivationHigh
USF1ActivationHigh
WT1ActivationHigh
TSC22D3ActivationHigh
RXRAActivationHigh
PPARGActivationHigh
PPARDActivationHigh
SREBF1ActivationHigh
FOXP3ActivationUnknown
FOXC2ActivationUnknown
FOXO1ActivationUnknown
ARActivationHigh

Repressive regulators: AHR, KLF2, PPARA, SMAD3, NR3C1, NR4A3, GLI1, SP6, ZNF174 (High confidence).

Drug & pharmacology data

TGFB1 is a known drug target with documented clinical development. The TGF-β signaling pathway (particularly ALK5/TGF-βR1) is actively targeted.

Targeting Molecules

Total identified in ChEMBL: 5 molecules

Top molecules by development phase:

MoleculeIDTypeHighest PhaseIndications
VACTOSERTIB (TEW-7197)CHEMBL3260567Small molecule (ALK5 inhibitor)Phase 2Pancreatic cancer, gastric cancer, colorectal cancer, lung cancer, melanoma, osteosarcoma, esophageal adenocarcinoma, myelodysplastic syndromes
GALUNISERTIB (LY-2157299)CHEMBL2364611Small molecule (ALK5 inhibitor)Phase 2Myelodysplastic syndromes, hepatocellular carcinoma, pancreatic cancer, glioblastoma, rectal cancer, ALS, renal fibrosis
FRESOLIMUMAB (GC-1008, GZ-402669)CHEMBL1743022Monoclonal antibody (TGF-β ligand neutralizer)Phase 2Idiopathic pulmonary fibrosis, focal segmental glomerulosclerosis, systemic sclerosis, glioma, mesothelioma, osteogenesis imperfecta

Clinical Trials

Top 20 from 50+ total trials (all three drugs combined):

GALUNISERTIB (22 trials):

  1. NCT02008318 | Phase 2/3 | Completed | Galunisertib in myelodysplastic syndromes
  2. NCT07321860 | Phase 2/3 | Not Yet Recruiting | Galunisertib + nerandomilast in ALS
  3. NCT01246986 | Phase 2 | Completed | LY2157299 in hepatocellular carcinoma
  4. NCT02178358 | Phase 2 | Completed | LY2157299 in advanced HCC
  5. NCT02452008 | Phase 2 | Active Not Recruiting | Galunisertib + enzalutamide in castration-resistant prostate cancer
  6. NCT02688712 | Phase 2 | Active Not Recruiting | Galunisertib in rectal cancer
  7. NCT04605562 | Phase 2 | Not Yet Recruiting | Galunisertib in nasopharyngeal cancer
  8. NCT02423343 | Phase 1/2 | Completed | Galunisertib + nivolumab in advanced solid tumors/NSCLC/HCC
  9. NCT01220271 | Phase 1/2 | Completed | LY2157299 + temozolomide-radiotherapy in malignant glioma
  10. NCT01373164 | Phase 1/2 | Completed | LY2157299 in metastatic/pancreatic cancer

VACTOSERTIB (19 trials): 11. NCT05588648 | Phase 1/2 | Recruiting | Vactosertib in osteosarcoma 12. NCT06044311 | Phase 2 | Recruiting | Vactosertib + chemoradiotherapy in esophageal adenocarcinoma 13. NCT03698825 | Phase 1/2 | Completed | TEW-7197 + paclitaxel in gastric cancer 14. NCT03724851 | Phase 1/2 | Completed | Vactosertib + pembrolizumab in colorectal/gastric cancer 15. NCT03732274 | Phase 1/2 | Completed | Vactosertib + durvalumab in advanced NSCLC 16. NCT03802084 | Phase 1/2 | Completed | Vactosertib + imatinib in advanced desmoid tumors 17. NCT03666832 | Phase 1/2 | Unknown | TEW-7197 + FOLFOX in metastatic pancreatic cancer 18. NCT04893252 | Phase 2 | Unknown | Vactosertib + durvalumab in gastric cancer 19. NCT04656002 | Phase 2 | Unknown | Vactosertib + paclitaxel + ramucirumab in gastric adenocarcinoma

FRESOLIMUMAB (9 trials): 20. NCT02581787 | Phase 1/2 | Completed | SABR-ATAC: Fresolimumab + stereotactic ablative radiotherapy in early NSCLC

Pharmacogenomics

Limited pharmacogenomics data available in current databases for TGFB1-specific associations. No established TGFB1 variant-specific dosing guidelines identified. Published literature describes:

  • TGFB1 polymorphisms: C-509T and T869C variants associated with baseline TGF-β levels and immune response, potentially affecting drug efficacy
  • Response prediction: Emerging biomarkers include TGF-β signaling pathway activation status (SMAD2/3 phosphorylation), ALK5 expression levels, and fibrotic/epithelial-mesenchymal transition (EMT) gene signatures
  • Drug-specific PK/PD: No major pharmacogenetic subgrouping required for current TGFB1-targeting drugs; dose adjustments primarily based on tolerability and organ function (hepatic/renal)

Expression profiles

Tissue and Cell Type Expression (Bgee)

TGFB1 shows ubiquitous expression across tissues with high signal in immune cells and vascular tissues.

Top 30 tissues/cell types by expression score:

RankEntityTypeExpression ScoreQuality
1GranulocyteCell type99.08Gold
2MonocyteCell type98.51Gold
3LeukocyteCell type98.47Gold
4Mononuclear cellCell type98.43Gold
5Stromal cell of endometriumCell type98.27Gold
6Ascending aortaTissue96.77Gold
7Thoracic aortaTissue96.75Gold
8Descending thoracic aortaTissue96.68Gold
9Right coronary arteryTissue96.46Gold
10SpleenTissue96.43Gold
11Lower esophagus mucosaTissue96.32Gold
12Right lungTissue95.95Gold
13EndocervixTissue95.60Gold
14BloodTissue95.46Gold
15AortaTissue95.39Gold
16Upper lobe of left lungTissue95.20Gold
17Left coronary arteryTissue95.04Gold
18Bone marrow cellCell type94.85Gold
19EctocervixTissue94.61Gold
20Coronary arteryTissue94.59Gold
21Upper lobe of lungTissue94.55Gold
22Popliteal arteryTissue94.50Gold
23Tibial arteryTissue94.50Gold
24Mucosa of stomachTissue93.90Gold
25Body of uterusTissue93.65Gold
26Lymph nodeTissue93.14Gold
27Omental fat padTissue92.73Gold
28PeritoneumTissue92.67Gold
29Metanephros cortex (kidney)Tissue92.50Gold
30Left uterine tubeTissue92.39Gold

Summary statistics:

  • 204/272 conditions show present expression
  • Average expression score: 82.96
  • Expression breadth: Ubiquitous (all surveyed tissues)

Pattern: Strong enrichment in immune cell populations (granulocytes > monocytes > leukocytes) and vascular endothelium (aortic/coronary arteries). Consistent moderate-to-high expression across blood, lymphoid tissues, and mucosal epithelium.

Single-Cell Expression (SCXA)

TGFB1 is characterized as a marker gene in 10 experiments spanning 259 cell clusters.

  • Max mean expression: 4451.79
  • Average mean expression: 175.59
  • Marker status: Present in all 10 analyzed experiments

Notable datasets:

  • E-ANND-5: Mapping developing human immune system (911,873 cells) — immune cell-enriched
  • E-GEOD-139324: Head and neck cancer immune landscape (204,315 cells) — tumor-immune microenvironment
  • E-GEOD-135922: Human retinal pigment epithelium and choroid (55,571 cells) — eye tissue with immune/fibroblast presence
  • E-MTAB-8205: hPSC-derived endothelial-to-haematopoietic transition (25,764 cells) — developmental pathway
  • E-MTAB-8911: GVHD T-lymphocytes (19,075 cells) — expanded T-cell clones
  • E-GEOD-106540: CD4+ cytotoxic T lymphocyte precursors (2,244 cells) — rare immune subset

Cell type pattern: Predominantly expressed in immune cells (T cells, B cells, myeloid cells) and stromal/fibroblast populations; consistent with growth factor and immune regulation functions.

Disease associations

Mendelian / Monogenic Diseases

Disease NameDisease IDInheritance PatternEvidence Level
Camurati-Engelmann diseaseOMIM:131300 / Orphanet:1328 / Mondo:0007542Autosomal dominantDefinitive (Ambry, G2P); Strong (Genomics England, Labcorp, PanelApp Australia)
Inflammatory bowel disease, immunodeficiency, and encephalopathyOMIM:618213 / Orphanet:565788 / Mondo:0032601Autosomal recessiveLimited (Ambry); Strong (Labcorp); Moderate (PanelApp Australia)
Cystic fibrosis (modifier gene)Orphanet:586 / Mondo:0009061Autosomal recessiveSupportive
Meckel syndrome, type 10Mondo:0013609VariableClinVar-derived
IL10-related early-onset inflammatory bowel diseaseMondo:0016542Autosomal recessiveClinVar-derived

Phenotype Associations (Top 30 HPO Terms)

HPO IDPhenotypeHPO IDPhenotype
HP:0000006Autosomal dominant inheritanceHP:0002240Hepatomegaly
HP:0000007Autosomal recessive inheritanceHP:0002315Headache
HP:0002024MalabsorptionHP:0002384Focal impaired awareness seizure
HP:0002059Cerebral atrophyHP:0002515Waddling gait
HP:0002099AsthmaHP:0002570Steatorrhea
HP:0002105HemoptysisHP:0002595Ileus
HP:0002110BronchiectasisHP:0002613Biliary cirrhosis
HP:0002188Delayed CNS myelinationHP:0002650Scoliosis
HP:0002205Recurrent respiratory infectionsHP:0002652Skeletal dysplasia
HP:0001298EncephalopathyHP:0003034Diaphyseal sclerosis
HP:0001324Muscle weaknessHP:0003565Elevated erythrocyte sedimentation rate
HP:0001376Limitation of joint mobilityHP:0005464Craniofacial osteosclerosis
HP:0001392Abnormality of the liverHP:0011001Increased bone mineral density
HP:0001394CirrhosisHP:0006532Recurrent pneumonia
HP:0001508Failure to thriveHP:0100759Clubbing of fingers

Complex-Disease / GWAS Associations (Top 19)

Trait/DiseaseMapped GeneChrP-valueStudy ID
Coronary artery diseaseTGFB1191e-26GCST010866_163
Coronary artery diseaseTGFB1192e-17GCST005195_133
Coronary artery diseaseTGFB1194e-17GCST005194_75
Coronary artery diseaseTGFB1197e-15GCST005196_248
Coronary artery diseaseTGFB1194e-16GCST005196_249
Aspartate aminotransferase levelsCCDC97, TGFB1192e-25GCST90013664_34
Pulse pressureTGFB1198e-11GCST007269_321
HematuriaCCDC97, TGFB1191e-11GCST008613_2
Platelet countCCDC97, TGFB1192e-11GCST90002402_208
Alanine aminotransferase levelsCCDC97, TGFB1194e-09GCST90013663_33
Coronary artery diseaseTGFB1194e-08GCST004787_13
Hematuria (moderate to severe)CCDC97, TGFB1192e-09GCST008617_4
Coronary artery diseaseTGFB1192e-08GCST007990_15
Preterm birth (maternal effect)TGFB1195e-07GCST004898_3
Colorectal cancerTMEM91194e-07GCST003494_2
Type 2 diabetesTGFB1191e-06GCST008114_4
Hematuria (mild)CCDC97, TGFB1199e-08GCST008618_1
Colorectal cancer or advanced adenomaTMEM91191e-06GCST007856_118
Colorectal cancerTMEM91191e-08GCST002454_12

Structured Data Sources

Generated with Claude Haiku 4.5 + BioBTree MCP, drawing on data BioBTree aggregates from 46 biological databases. Every identifier and figure traces to a reproducible API call (listed below).

Further analyze this answer or run your own queries with BioBTree MCP.

Datasets: alphafold, alphamissense, antibody, bgee, biogrid_interaction, cathgene3d, ccds, chembl_molecule, chembl_target, chitars, clinical_trials, clinvar, collectri, diamond_similarity, drugbank, ensembl, entrez, esm2_similarity, exon, gencc, go, gtex, gwas, hgnc, hpa, hpo, intact, interpro, mim, mondo, msigdb, orphanet, pdb, pfam, pharmgkb, pirsf, prints, reactome, refseq, scxa, scxa_expression, smart, spliceai, string_interaction, transcript, uniprot
Generated: 2026-05-26 — For the latest data, query BioBTree directly via MCP or API.
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