glial cell line-derived neurotrophic factor (Amgen)

Glial cell line-derived neurotrophic factor (GDNF) is a naturally occurring protein and member of the transforming growth factor-beta superfamily. It potently promotes the survival and differentiation of various neuronal populations, especially dopaminergic neurons in the central nervous system. GDNF acts by binding to a receptor complex composed of GFRα1 and RET receptor tyrosine kinase, activating intracellular signaling pathways that support neuron survival and regeneration[7][10]. Because it is a large protein that does not cross the blood-brain barrier, therapeutic use requires direct delivery to brain tissue or gene therapy approaches. Recombinant GDNF has been investigated as a potential disease-modifying treatment for Parkinson’s disease due to its ability to protect and restore dopamine-producing neurons[2][3][4][5]. Clinical trials have used both direct infusion into brain regions such as the putamen and gene therapy vectors (e.g., AAV2-GDNF) for localized expression[1][4][9]. While some studies have shown biological effects on brain cells, clinical efficacy remains inconclusive; further research is ongoing. **Sub-categories:** * **Modality:** Gene therapy * **Payload:** human glial cell line-derived neurotrophic factor gene * **Vector Type:** adeno-associated viral vector serotype 2 (AAV2) * **Comment:** Used in investigational therapies for Parkinson's disease[1][4]

Other names

glial cell line derived neurotrophic factorglial cell-derived neurotrophic factorGDNF protein