Review Article
Gene Therapy for Parkinson's Disease
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] |
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AADC, aromatic amino acid decarboxylase; GAD, glutamic acid decarboxylase; GCH-1, GTP-cyclohydrolase-1; GDNF, glial cell line-derived neurotrophic factor; TH, tyrosine hydroxylase.
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