Protein / target

Retinoic acid receptor alpha

RARAP10276Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
7
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

MRNA regulatory element binding translation repressor activity

Primary system

Nervous system

Strongest disease association

neoplasm

Genetic evidence · score 0.19

Therapeutic maturity

Clinically validated target

7 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

7 approved · 4 in clinical development

30 linked trials

Research activity

Emerging research

1 papers · latest 2020

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Receptor for retinoic acid (PubMed:16417524, PubMed:19850744, PubMed:20215566, PubMed:21152046, PubMed:37478846). Retinoic acid receptors bind as heterodimers to their target response elements in response to their ligands, all-trans or 9-cis retinoic acid, and regulate gene expression in various biological processes (PubMed:21152046, PubMed:28167758, PubMed:37478846). The RXR/RAR heterodimers bind to the retinoic acid response elements (RARE) composed of tandem 5'-AGGTCA-3' sites known as DR1-DR5 (PubMed:19398580, PubMed:28167758). In the absence of ligand, the RXR-RAR heterodimers associate with a multiprotein complex containing transcription corepressors that induce histone deacetylation, chromatin condensation and transcriptional suppression (PubMed:16417524). On ligand binding, the corepressors dissociate from the receptors and associate with the coactivators leading to transcriptional activation (PubMed:19850744, PubMed:20215566, PubMed:37478846, PubMed:9267036). Formation of a complex with histone deacetylases might lead to inhibition of RARE DNA element binding and to transcriptional repression (PubMed:28167758). Transcriptional activation and RARE DNA element binding might be supported by the transcription factor KLF2 (PubMed:28167758). RARA plays an essential role in the regulation of retinoic acid-induced germ cell development during spermatogenesis (By similarity). Has a role in the survival of early spermatocytes at the beginning prophase of meiosis (By similarity). In Sertoli cells, may promote the survival and development of early meiotic prophase spermatocytes (By similarity). In concert with RARG, required for skeletal growth, matrix homeostasis and growth plate function (By similarity). Together with RXRA, positively regulates microRNA-10a expression, thereby inhibiting the GATA6/VCAM1 signaling response to pulsatile shear stress in vascular endothelial cells (PubMed:28167758). In association with HDAC3, HDAC5 and HDAC7 corepressors, plays a role in the repression of microRNA-10a and thereby promotes the inflammatory response (PubMed:28167758)

Subcellular location

NucleusCytoplasm
Domains and Gene Ontology detail (74)

Domains & features

NR LBD

Gene Ontology

  • Cchromatin
  • Ccytoplasm
  • Ccytosol
  • Cdendrite
  • Cnucleolus
  • Cnucleoplasm
  • Cnucleus
  • Cperinuclear region of cytoplasm
  • Cplasma membrane
  • Cprotein-containing complex
  • CRNA polymerase II transcription regulator complex
  • Falpha-actinin binding

462 aa · 51 kDa · 3 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Transcriptional regulationUniProt · GO · ReactomeNuclear receptor signallingGO · ReactomeImmune signallingUniProt · GOKinase signallingGOApoptosis & cell deathGO
View supporting evidence

Transcriptional regulation

  • ·Receptor for retinoic acid (PubMed:16417524, PubMed:19850744, PubMed:20215566, PubMed:21…
  • ·RNA polymerase II transcription regulator complex
  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor activity, RNA polymerase II-specific

Nuclear receptor signalling

  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor activity, RNA polymerase II-specific
  • ·nuclear receptor activity
  • ·Nuclear Receptor transcription pathway

Immune signalling

  • ·Receptor for retinoic acid (PubMed:16417524, PubMed:19850744, PubMed:20215566, PubMed:21…
  • ·positive regulation of interleukin-13 production
  • ·positive regulation of interleukin-4 production
  • ·positive regulation of interleukin-5 production

Kinase signalling

  • ·protein kinase A binding
  • ·protein kinase B binding
  • ·protein phosphorylation

Apoptosis & cell death

  • ·regulation of apoptotic process
View underlying pathways (6)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

NCOR1NCOR2RXRAPMLZBTB16NCOA1RXRBNCOA2RARBRXRGRARA

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

2

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

Isotretinoin
ApprovedAgonist

Retinoic acid receptor agonist

Appears in clinical studies involving acne, acne, glioma, renal cell carcinoma

Acts on a complex — shared with RARB, RARG · 1 of 3 recorded protein targets — narrow recorded profile

Tretinoin
ApprovedAgonist

Retinoic acid receptor agonist

Appears in clinical studies involving hyperpigmentation of the skin, acne, acute promyelocytic leukemia, freckles

Acts on a complex — shared with RARB, RARG · 1 of 3 recorded protein targets — narrow recorded profile

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

neoplasm0.19

Genetic · overall 0.51

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

acne1.00

Clinical · overall 0.61

psoriasis0.97

Clinical · overall 0.59

acute promyelocytic leukemia0.95

Clinical · overall 0.61

psoriasis vulgaris0.94

Clinical · overall 0.58

hyperpigmentation of the skin0.89

Clinical · overall 0.54

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

freckles0.52

Clinical

acute myeloid leukemia0.51

Literature

Kaposi's sarcoma0.47

Clinical

Eczematoid dermatitis0.46

Clinical

Show all associations
acne0.61
acute promyelocytic leukemia0.61
psoriasis0.59
psoriasis vulgaris0.58
hyperpigmentation of the skin0.54
freckles0.52
acute myeloid leukemia0.51
neoplasm0.51
Kaposi's sarcoma0.47
Eczematoid dermatitis0.46

Open Targets ranks 1,955 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 11 total

TRETINOINApproval

hyperpigmentation of the skin · acne · acute promyelocytic leukemia

ALITRETINOINApproval

Kaposi's sarcoma · Eczematoid dermatitis · Kaposi's sarcoma

NRX195183Phase 2

acute promyelocytic leukemia · plasma cell myeloma · lymphoma

TAMIBAROTENEPhase 3

myelodysplastic syndrome · Alzheimer disease · tropical spastic paraparesis

ETRETINATEApproval

psoriasis · keratosis

ISOTRETINOINApproval

acne · acne · glioma

ADAPALENEApproval

acne · acne · molluscum contagiosum

ACITRETINApproval

psoriasis · psoriasis vulgaris · hand eczema

TAZAROTENEApproval

psoriasis vulgaris · acne · psoriasis

MOFAROTENEPhase 1

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC high confPR · LiteraturePR · UniProt UbiquitinationPR · Database UbiquitinationPR · Small Molecule Binder

Safety liabilities

regulation of transcription factor activityreceptor bindingNegative effects on testicular histology and spermatogenesis

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

COMPLETED · via Isotretinoin · NCT00499616

WITHDRAWN · via Isotretinoin · NCT01319838

COMPLETED · via Isotretinoin · NCT00098020

COMPLETED · via Tretinoin · NCT00000621

ClinicalTrials.gov via the drug-target graph.

Research activity

1 papers · to 2020

Papers linked directly to this protein. This is the protein's own literature — descriptor-derived papers are kept separate below.

Most cited

Recent

Europe PMC papers linked directly to this protein.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

acute promyelocytic leukemiaWell supported
0.76
agreement 0.600.91
Clinical83%Literature17%

Open Targets aggregate 0.61 · 2 independent evidence families

acneWell supported
0.75
agreement 0.590.91
Clinical99%Literature1%

Open Targets aggregate 0.61 · 2 independent evidence families

acute myeloid leukemiaModerately supported
0.74
agreement 0.620.86
Clinical49%Somatic mutation38%Literature13%

Open Targets aggregate 0.51 · 3 independent evidence families

psoriasisModerately supported
0.73
agreement 0.570.89
Clinical99%Literature2%

Open Targets aggregate 0.59 · 2 independent evidence families

psoriasis vulgarisModerately supported
0.71
agreement 0.560.86
Clinical100%Literature1%

Open Targets aggregate 0.58 · 2 independent evidence families

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

57

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 2 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. Label change2026-06-16

    Label change: ISOTRETINOIN (ANDA213571)

    fda · regulatory · fda · via Isotretinoin

  2. Label change2026-06-12

    Label change: ISOTRETINOIN (NDA211913)

    fda · regulatory · fda · via Isotretinoin

  3. Label change2026-06-12

    Label change: ISOTRETINOIN (NDA021951)

    fda · regulatory · fda · via Isotretinoin

  4. Regulatory approval2026-06-11

    Approval: TRETINOIN (ANDA214590)

    fda · regulatory · fda · via Tretinoin

  5. Supplemental approval2026-02-09

    Supplemental approval: ISOTRETINOIN (NDA211913)

    fda · regulatory · fda · via Isotretinoin

  6. Supplemental approval2026-02-09

    Supplemental approval: ISOTRETINOIN (ANDA213571)

    fda · regulatory · fda · via Isotretinoin

  7. Supplemental approval2026-02-09

    Supplemental approval: ISOTRETINOIN (NDA021951)

    fda · regulatory · fda · via Isotretinoin

  8. New publication2024-07-17
    Differentiation of SH-SY5Y neuroblastoma cells using retinoic acid and BDNF: a model for neuronal and synaptic differentiation in neurodegeneration.

    In vitro cellular & developmental biology. Animal · 2024 · 20 citations · Europe PMC · via Tretinoin

  9. Label change2024-06-28

    Label change: ISOTRETINOIN (NDA021951)

    fda · regulatory · fda · via Isotretinoin

  10. New publication2024-01-30
    Guidelines of care for the management of acne vulgaris.

    Journal of the American Academy of Dermatology · 2024 · 236 citations · Europe PMC · via Isotretinoin

  11. New publication2024-01-01
    Risk of Suicide and Psychiatric Disorders Among Isotretinoin Users: A Meta-Analysis.

    JAMA dermatology · 2024 · 24 citations · Europe PMC · via Isotretinoin

  12. New publication2023-11-21
    Effect of isotretinoin on CYP2D6 and CYP3A activity in patients with severe acne.

    British journal of clinical pharmacology · 2024 · 1 citation · Europe PMC · via Isotretinoin

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.