Protein / target
Dystrophin
Protein at a glance
Biological role
Structural constituent of cytoskeleton
Primary system
Nervous system
Strongest disease association
neuromuscular disease caused by qualitative or quantitative defects of dystrophin
Therapeutic maturity
Clinically validated target
Druggability
Antibody
Clinical development
5 approved · 2 in clinical development
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
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.
View supporting evidenceHide supporting evidence
Synaptic signalling
- ·Anchors the extracellular matrix to the cytoskeleton via F-actin. Ligand for dystroglyca…
- ·Postsynaptic cell membrane
- ·postsynaptic membrane
- ·synapse
Muscle contraction
- ·myosin binding
- ·cardiac muscle contraction
- ·regulation of cardiac muscle contraction by regulation of the release of sequestered cal…
- ·regulation of skeletal muscle contraction
Ion channel gating
- ·regulation of calcium ion transmembrane transport
- ·regulation of sodium ion transmembrane transport
Cell adhesion
- ·Anchors the extracellular matrix to the cytoskeleton via F-actin. Ligand for dystroglyca…
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.
Drugs targeting this protein
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.
Dystrophin exogenous gene
Appears in clinical studies involving Becker muscular dystrophy, Duchenne muscular dystrophy, Duchenne muscular dystrophy, Duchenne muscular dystrophy
ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.
Translational evidence
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.
Highest-confidence therapeutic associations
Diseases where a drug acting on this target has already reached clinical development.
Highest overall evidence
Remaining associations by Open Targets' aggregated evidence score.
Show all associationsHide all associations
Open Targets ranks 4,815 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 · 8 total
Becker muscular dystrophy · Duchenne muscular dystrophy · Duchenne muscular dystrophy
Duchenne muscular dystrophy
Duchenne muscular dystrophy · Duchenne muscular dystrophy
muscular dystrophy · Duchenne muscular dystrophy · Duchenne muscular dystrophy
muscular dystrophy · Duchenne muscular dystrophy · Duchenne muscular dystrophy
muscular dystrophy · Duchenne muscular dystrophy · Duchenne muscular dystrophy
Duchenne muscular dystrophy · Duchenne muscular dystrophy
muscular dystrophy · Duchenne muscular dystrophy · Duchenne muscular dystrophy
Tractability
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.
ClinicalTrials.gov via the drug-target graph.
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.
Forefront confidence
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.
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
Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.
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.