Protein / target
Dystrophin
Protein at a glance
Biological role
Structural constituent of cytoskeleton
Strongest disease association
Neuromuscular disease caused by qualitative or quantitative defects of dystrophin
Therapeutic position
Established drug target
Research activity
Emerging research
Derived from structured UniProt, Open Targets and literature data on this page.
Protein profile
Canonical identity and biological annotation from UniProt.
Function overview
Anchors the extracellular matrix to the cytoskeleton via F-actin.
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Anchors the extracellular matrix to the cytoskeleton via F-actin. Ligand for dystroglycan. Component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction (NMJ) and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma. Also implicated in signaling events and synaptic transmission
Subcellular location
Domains and Gene Ontology detail (55)Hide
Domains & features
Gene Ontology
- Ccell surface
- Ccell-substrate junction
- Ccostamere
- Ccytoskeleton
- Ccytosol
- Cdystrophin-associated glycoprotein complex
- Cfilopodium
- Cfilopodium membrane
- Cmembrane raft
- Cneuron projection terminus
- Cnucleus
- Cplasma membrane
Biological roles
What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.
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Synaptic signalling
- ·Anchors the extracellular matrix to the cytoskeleton via F-actin. Ligand for dystroglyca…
- ·Postsynaptic cell membrane
- ·postsynaptic membrane
- ·synapse
Ion channel gating
- ·regulation of calcium ion transmembrane transport
- ·regulation of sodium ion transmembrane transport
Muscle contraction
- ·cardiac muscle contraction
- ·regulation of cardiac muscle contraction by regulation of the release of sequestered cal…
- ·regulation of skeletal muscle contraction
- ·regulation of skeletal muscle contraction by regulation of release of sequestered calciu…
View underlying pathways (3)Hide underlying pathways
Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.
Interaction neighbourhood
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.
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.
Approved medicines with mapped indications
Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.
5 medicines meet Open Targets' target-level approved-medicine definition; the 1 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.
Drugs targeting this protein
How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.
Dystrophin exogenous gene
Indicated for Muscular Dystrophy, Duchenne
ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.
Translational evidence
Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.
Strongest disease associations · via encoding gene DMD
Gene-level evidence surfaced through the gene DMDthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.
View evidence synthesis (5)Hide
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 per-type scores above show the calculation.
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Drug development
8 compounds recorded · 5 approved · 2 in clinical development · 1 earlier-stage
View all recorded compounds (8)Hide
Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.
Tractability
Antibodies — Emerging
Protein degraders — Emerging
View underlying tractability evidence (4)Hide
Raw Open Targets tractability assessment buckets, by modality.
Clinical trials
Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.
ClinicalTrials.gov via the drug-target graph.
What's happening now
Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.
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.
Research activity
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.