Table 1.
An overview of the therapeutic approaches used in current clinical trials of gene therapy for Parkinson's disease.
| Therapeutic approach | Vector | Clinical trials |
|---|---|---|
| Increased dopamine biosynthesis | ||
| (i) AADC alone | Adeno-associated virus | Phase I [13, 60] |
| (ii) AADC, TH, and GCH-1 | Lentivirus | Phase I/ II in progress [54] |
| Modulation of excitatory and inhibitory basal ganglia activity | ||
| (i) GAD | Adeno-associated virus | Phase I and II [61, 62] |
| Neurotrophic support | ||
| (i) GDNF | Adeno-associated virus | License obtained to develop, no published clinical trials [63] |
| (ii) Neurturin | Adeno-associated virus | Phase I and II [12, 59] |
AADC, aromatic amino acid decarboxylase; GAD, glutamic acid decarboxylase; GCH-1, GTP-cyclohydrolase-1; GDNF, glial cell line-derived neurotrophic factor; TH, tyrosine hydroxylase.