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]

AADC, aromatic amino acid decarboxylase; GAD, glutamic acid decarboxylase; GCH-1, GTP-cyclohydrolase-1; GDNF, glial cell line-derived neurotrophic factor; TH, tyrosine hydroxylase.