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Target intelligence / Profile preview
PML-retinoic acid receptor alpha fusion protein (PML-RARA)
- Target
- PML-RARA
- Molecular classification
- Transcription factor, Fusion protein, Oncoprotein
01
Overview
The PML-retinoic acid receptor alpha (PML-RARA) fusion protein is a chimeric oncoprotein resulting from the t(15;17)(q24;q21) chromosomal translocation found in acute promyelocytic leukemia (APL). This fusion protein acts as an aberrant transcription factor that represses target genes critical for myeloid differentiation by recruiting co-repressors such as SMRT, N-CoR, RXR, TBLR1, and histone deacetylases/methyltransferases. It also disrupts the formation/function of PML nuclear bodies. The fusion protein is targeted therapeutically by arsenic trioxide and all-trans retinoic acid, which induce degradation and overcome transcriptional repression, respectively, leading to differentiation of leukemic cells.
- Other names
- PML::RARAPML/RAR-alphaPromyelocytic leukemia protein/retinoic acid receptor alpha fusion protein
02
Mechanism of action
Arsenic trioxide induces degradation of PML portion; All-trans retinoic acid overcomes transcriptional repression imposed by the fusion protein.
03
Biological functions
Transcriptional regulationRepression of myeloid differentiationDisruption of nuclear bodiesDNA binding
04
Disease associations
Acute promyelocytic leukemia (APL)Cancer
05
Safety considerations
Differentiation syndrome (ATRA syndrome)QT prolongation (Arsenic trioxide)LeukocytosisCoagulopathy
06
Interacting drugs
Arsenic trioxide
1 more in the full profile.
07
Biomarkers
PML-RARA transcript levelsMinimal residual disease (MRD)
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