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Target intelligence / Profile preview
Glial Cell Line-Derived Neurotrophic Factor (GDNF)
- Target
- GDNF
- Molecular classification
- Growth Factor, TGF-beta superfamily ligand, GFL (GDNF Family Ligand)
01
Overview
Glial cell line-derived neurotrophic factor (GDNF) is a small, glycosylated protein that plays a critical role in the survival, development, and maintenance of various types of neurons, particularly dopaminergic neurons. It signals through the GFRα1/RET receptor complex and has been investigated as a therapeutic target for neurodegenerative diseases like Parkinson's disease. Beyond neuronal survival, GDNF modulates sodium channel expression and glial activation, suggesting potential applications in neuropathic pain management. Expression patterns include dopamine-rich brain regions and gliomas.
02
Mechanism of action
Binds to GFRα1 co-receptor, which then activates the RET tyrosine kinase receptor. Alternatively, in the absence of RET, it can signal via NCAM, activating Fyn/FAK kinases.
03
Biological functions
Neuron survivalNeuron growthNeuron maintenanceNeuron regenerationKidney developmentSodium channel modulationGlial activation modulationNeuroinflammation modulationCell proliferation (in gliomas)
04
Disease associations
Parkinson's diseaseNeurodegenerative diseasesRenal agenesisNeuropathic painGlioma
05
Safety considerations
Challenges with protein delivery across the blood-brain barrierClinical trials showed limited efficacy despite biological activityPotential for off-target effects due to widespread expression
06
Interacting drugs
Vitamin D
1 more in the full profile.
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