pGDNF_1b

pGDNF_1b is a DNA plasmid encoding the human glial cell line–derived neurotrophic factor (GDNF) splice variant 1, delivered as a compacted DNA nanoparticle for gene therapy. It uses the polyubiquitin C (UbC) promoter active primarily in glia, and its cassette design includes flanking scaffold/matrix attachment elements and a CpG-depleted prokaryotic domain to enhance expression. Intracerebral administration of pGDNF_1b nanoparticles results in sustained, long-term GDNF expression at biologically active levels, aiming for neuroprotection in models of Parkinson’s disease. Its mechanism is gene-mediated overexpression of GDNF to support neuron survival and function, particularly in the striatum.

Other names

pGDNF nanoparticlesGDNF plasmid nanoparticlesnano-GDNFGDNF-pDNA nanoparticles