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glial cell line-derived neurotrophic factor (MedGenesis Therapeutix Inc.)
- Development stage
- Unknown
- Lead developer
- MedGenesis Therapeutix
- Modality
- Cytokines & Interferons → Recombinant Proteins and Enzymes
- Administration
- Intracerebral, Intraventricular, Intraparenchymal
01
Overview
Glial cell line-derived neurotrophic factor (GDNF) is a small protein and member of the transforming growth factor-beta (TGF-β) superfamily, structurally classified as a cysteine knot protein. It acts as a potent survival and trophic factor for various neuronal populations, especially midbrain dopaminergic neurons, noradrenergic neurons of the locus coeruleus, and spinal motor neurons. GDNF signals primarily through binding to its co-receptor GFRα1 (GDNF family receptor alpha 1), which then activates the RET receptor tyrosine kinase. This signaling promotes neuronal survival, differentiation, maintenance, and regeneration. In addition to its role in the nervous system—including development and maintenance of sensory, sympathetic, enteric neurons—GDNF also has functions outside the nervous system such as promoting ureteric branching during kidney development. Preclinical studies have shown that GDNF can restore function in animal models of Parkinson’s disease by supporting dopaminergic neuron survival; however, clinical trials in humans have not demonstrated significant efficacy for Parkinson’s disease despite evidence of biological activity[2][4][5][6][7].
- Other names
- glial cell line-derived neurotrophic factor
02
Targets
GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)
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