OncoSignal
Live from Open Targets Platform, fetched 2026-11-11 22:12 UTC. Every row links to its source record.

ASXL1 ASXL transcriptional regulator 1

ENSG00000171456 · protein coding · Open Targets profile

Probable Polycomb group (PcG) protein involved in transcriptional regulation mediated by ligand-bound nuclear hormone receptors, such as retinoic acid receptors (RARs) and peroxisome proliferator-activated receptor gamma (PPARG) (PubMed:16606617). Acts as a coactivator of RARA and RXRA through association with NCOA1 (PubMed:16606617). Acts as a corepressor for PPARG and suppresses its adipocyte differentiation-inducing activity (By similarity). Non- catalytic component of the PR-DUB complex, a complex that specifically mediates deubiquitination of histone H2A monoubiquitinated at 'Lys-119' (H2AK119ub1) (PubMed:20436459, PubMed:30664650, PubMed:36180891). Acts as a sensor of N(6)-methyladenine methylation on DNA (6mA): recognizes and binds 6mA DNA, leading to its ubiquitination and degradation by TRIP12, thereby inactivating the PR-DUB complex and regulating Polycomb silencing (PubMed:30982744). The PR-DUB complex is an epigenetic regulator of gene expression and acts as a transcriptional coactivator, affecting genes involved in development, cell communication, signaling, cell proliferation and cell viability (PubMed:30664650, PubMed:36180891). ASXL1, ASXL2 and ASXL3 function redundantly in the PR-DUB complex (By similarity) (PubMed:30664650). The ASXL proteins are essential for chromatin recruitment and transcriptional activation of associated genes (By similarity). ASXL1 and ASXL2 are important for BAP1 protein stability (PubMed:30664650). Together with BAP1, negatively regulates epithelial-mesenchymal transition (EMT) of trophoblast stem cells during placental development by regulating genes involved in epithelial cell integrity, cell adhesion and cytoskeletal organization (PubMed:34170818). {ECO:0000250|UniProtKB:P59598, ECO:0000269|PubMed:16606617, ECO:0000269|PubMed:20436459, ECO:0000269|PubMed:30664650, ECO:0000269|PubMed:30982744, ECO:0000269|PubMed:34170818, ECO:0000269|PubMed:36180891}.

In 11 of 15 cancers, the evidence for ASXL1 comes mainly from inherited variants

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)
00.250.50.751myelodysplastic syndromemyelodysplastic syndromemyelodysplastic syndrome: somatic 0.60myelodysplastic syndrome: germline 0.90acute myeloid leukemiaacute myeloid leukemiaacute myeloid leukemia: somatic 0.75acute myeloid leukemia: germline 0.74myeloid neoplasmmyeloid neoplasmmyeloid neoplasm: somatic 0.61myeloid neoplasm: germline 0.91myeloid leukemiamyeloid leukemiamyeloid leukemia: germline 0.96hematopoietic and lymphoid system neopl…hematopoietic and lymphoid system neoplasmhematopoietic and lymphoid system neoplasm: germline 0.92lymphoid neoplasmlymphoid neoplasmlymphoid neoplasm: somatic 0.46lymphoid neoplasm: germline 0.80myeloproliferative disordermyeloproliferative disordermyeloproliferative disorder: somatic 0.46myeloproliferative disorder: germline 0.68Common Hematopoietic NeoplasmCommon Hematopoietic NeoplasmCommon Hematopoietic Neoplasm: germline 0.80primary myelofibrosisprimary myelofibrosisprimary myelofibrosis: germline 0.77juvenile myelomonocytic leukemiajuvenile myelomonocytic leukemiajuvenile myelomonocytic leukemia: somatic 0.46juvenile myelomonocytic leukemia: germline 0.60chronic myeloid leukemiachronic myeloid leukemiachronic myeloid leukemia: somatic 0.61chronic myeloid leukemia: germline 0.21hematopoietic and lymphoid cell neoplasmhematopoietic and lymphoid cell neoplasmhematopoietic and lymphoid cell neoplasm: germline 0.68chronic myelomonocytic leukemiachronic myelomonocytic leukemiachronic myelomonocytic leukemia: somatic 0.61chronic myelomonocytic leukemia: germline 0.19adult acute myeloid leukemiaadult acute myeloid leukemiaadult acute myeloid leukemia: germline 0.67B-cell chronic lymphocytic leukemiaB-cell chronic lymphocytic leukemiaB-cell chronic lymphocytic leukemia: somatic 0.52

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

PROTAC
UniProt Ubiquitination, Database Ubiquitination

gnomAD loss of function constraint: o/e 0.7291399836540222 (upper bound 0.8963599801063538). 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 ASXL1's approved or late-stage drugs in the last three years.

No terminated, withdrawn or suspended trials of ASXL1'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.