In vivo gene delivery of glial cell line--derived neurotrophic factor for Parkinson's disease.

Parkinson's disease (PD) is a progressive neurodegenerative disorder that affects approximately 1,000,000 Americans. The cause of the disease remains unknown. The histopathological hallmarks of the disease are dopaminergic striatal insufficiency secondary to a loss of dopaminergic neurons in the substantia nigra pars compacta and intracellular inclusion called Lewy bodies. Currently, only symptomatic treatment for PD is available. Although some treatments are efficacious for many years, all have significant limitations and new therapeutic approaches are needed. Gene therapy is ideal for delivering therapeutic molecules to site-specific regions of the central nervous system. Via gene therapy, a piece or pieces of DNA placed into a carrying vector encoding for a substance of interest can be introduced into specific cells. Although there are several ways that gene therapy can be applied for PD, this review focuses on in vivo gene delivery of glial cell line-derived neurotrophic factor (GDNF) as a neuroprotective strategy for PD.