Target intelligence / Profile preview

Runt-related Transcription Factor 1 (RUNX1)

Target
RUNX1
Molecular classification
Transcription factor, Runt-related transcription factor family, CBFα
01

Overview

Runt-related transcription factor 1 (RUNX1) is a master regulatory protein crucial for blood cell development but also involved more broadly across tissues including bone formation and heart tissue remodeling. Its dysregulation—by mutation or chromosomal rearrangement—is strongly linked to several cancers including acute myeloid leukemia. Experimental small-molecule inhibitors exist but none are yet clinically approved.

Other names
AML1CBFA2Acute Myeloid Leukemia 1 proteinCore-binding factor subunit alpha-2
02

Mechanism of action

Inhibition of RUNX1 transcriptional activity

03

Biological functions

Hematopoiesis regulationImmune modulationBone formationNeuronal developmentRegulation of gene expression
04

Disease associations

Acute Myeloid LeukemiaMyelodysplastic SyndromeBreast CancerAutoimmune DiseasesCardiac HypertrophyHeart Failure
05

Safety considerations

Potential for off-target effects due to broad role in gene regulationHematological toxicities
06

Interacting drugs

Ro24-7429

1 more in the full profile.

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