glial cell derived neurotrophic factor plasmid dna

Glial cell derived neurotrophic factor plasmid DNA is a **gene therapy approach** where a DNA plasmid encoding the human glial cell-derived neurotrophic factor (GDNF) gene is delivered to neural or glial cells to enable in situ production of GDNF protein. GDNF is a neurotrophic factor crucial for the survival and function of dopaminergic neurons, and its gene delivery by plasmid DNA aims to promote neuroprotection, regeneration, and functional recovery in neurodegenerative diseases, particularly Parkinson’s disease. In preclinical and early clinical research, GDNF gene plasmids are introduced via non-viral or viral methods, such as direct injection or device-mediated delivery, targeting affected areas of the brain. The locally expressed GDNF protein then acts on its receptors (GFRα1/RET complex) on neurons to activate neuroprotective intracellular pathways, enhance neuron survival, and support dopaminergic neuron function[1][4][9]. Developers have explored this technology to bypass the challenges of protein delivery due to the blood-brain barrier and protein instability.

Other names

GDNF plasmid DNAglial cell line-derived neurotrophic factor plasmid DNAGDNF gene plasmidGDNF pDNA