Indeed, in animal models of PD, GDNF has been shown to protect the dopaminergic nigrostriatal pathway from 6-OHDA or MPTP-induced degeneration when administered as a protein or gene therapy (4–7), and to restore the dopaminergic phenotype (i.e., striatal dopaminergic markers, such as tyrosine hydroxylase (TH) and the dopamine level) of the pathway after the degeneration in rodent and non-human primate models of PD (3, 8–11). Here, GDNF is linked to Parkinson disease.