OncoSignal
Live from Open Targets Platform, fetched 2026-11-12 00:36 UTC. Every row links to its source record.

SUFU SUFU negative regulator of hedgehog signaling

ENSG00000107882 · protein coding · Open Targets profile

Negative regulator in the hedgehog/smoothened signaling pathway that acts by sequestering the GLI (GI1, GLI2 and GLI3) transcription factors in the cytoplasm (PubMed:10559945, PubMed:10564661, PubMed:10806483, PubMed:12068298, PubMed:12975309, PubMed:15367681, PubMed:22365972, PubMed:24217340, PubMed:24311597, PubMed:27234298, PubMed:28965847, PubMed:35831023). When smoothened signaling is initiated, GLI transcription factors dissociate from SUFU, translocate to the nucleus and activate expression of target genes (PubMed:24311597, PubMed:28965847, PubMed:35831023). In absence of smoothened signaling, the SUFU-GLI3 complex is recruited to cilia, leading to the efficient processing of full-length GLI3 into transcription repressor GLI3R: SUFU participates to GLI3 processing by promoting recruitment of GSK3B to GLI3 (PubMed:24311597, PubMed:28965847). May also act as a negative regulator of beta-catenin signaling (By similarity). Required for normal embryonic development (By similarity). Required for the proper formation of hair follicles and the control of epidermal differentiation (By similarity). {ECO:0000250|UniProtKB:Q9Z0P7, ECO:0000269|PubMed:10559945, ECO:0000269|PubMed:10564661, ECO:0000269|PubMed:10806483, ECO:0000269|PubMed:12068298, ECO:0000269|PubMed:12975309, ECO:0000269|PubMed:15367681, ECO:0000269|PubMed:22365972, ECO:0000269|PubMed:24217340, ECO:0000269|PubMed:24311597, ECO:0000269|PubMed:27234298, ECO:0000269|PubMed:28965847, ECO:0000269|PubMed:35831023}.

In 10 of 15 cancers, the evidence for SUFU comes mainly from mutations found in tumours

Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.

Inherited (germline) variantsMutations in tumours (somatic)
00.250.50.751medulloblastomamedulloblastomamedulloblastoma: somatic 0.61medulloblastoma: germline 0.93nevoid basal cell carcinoma syndromenevoid basal cell carcinoma syndromenevoid basal cell carcinoma syndrome: germline 0.87familial meningiomafamilial meningiomafamilial meningioma: germline 0.63hereditary neoplastic syndromehereditary neoplastic syndromehereditary neoplastic syndrome: germline 0.91meningiomameningiomameningioma: somatic 0.46meningioma: germline 0.49skin basal cell carcinomaskin basal cell carcinomaskin basal cell carcinoma: somatic 0.61lung carcinomalung carcinomalung carcinoma: somatic 0.61esophageal adenocarcinomaesophageal adenocarcinomaesophageal adenocarcinoma: somatic 0.61hepatobiliary neoplasmhepatobiliary neoplasmhepatobiliary neoplasm: somatic 0.61nodular malignant melanomanodular malignant melanomanodular malignant melanoma: somatic 0.61Transitional MeningiomaTransitional MeningiomaTransitional Meningioma: somatic 0.61Chordoid MeningiomaChordoid MeningiomaChordoid Meningioma: somatic 0.61bile duct carcinomabile duct carcinomabile duct carcinoma: somatic 0.61superficial spreading melanomasuperficial spreading melanomasuperficial spreading melanoma: somatic 0.61skin carcinomaskin carcinomaskin carcinoma: somatic 0.61

Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.

Tractability

Small molecule
High-Quality Pocket
PROTAC
UniProt Ubiquitination, Database Ubiquitination

gnomAD loss of function constraint: o/e 0.11874999850988388 (upper bound 0.22303999960422516). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.

Who runs trials of these drugs

No named investigators on registry trials of SUFU's approved or late-stage drugs in the last three years.

No terminated, withdrawn or suspended trials of SUFU's drugs are registered

All 0 drugs and clinical candidates as a table

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.

DrugTypeHighest stageIndicationsSource records
No drugs or clinical candidates recorded.