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. 2018 Jun 13;8(2):195–215. doi: 10.3233/JPD-181331

Table 2.

NHP trials and clinical trials combining TH and AADC

Author Method Subjects Results Reference
During et al. (1998) Treatment with mix of AAV-TH and AAV-AADC in the striatum. MPTP-treated green monkeys (n = 12). Biochemically measured increase of dopamine production. No behavioral difference from non-transfected individuals. [38]
Muramatsu et al. (2002) Unilateral AAV induced expression of TH, GCH and AADC in the striatum. MPTP-treated marmosets (n = 4). Bilateral improvement of motor skills; improved fine motor skills. [48]
Sehara et al. (2017) Follow-up on Muramatsu et al. (2002). MPTP-treated marmoset (n = 1). Continuous effect on motor skills. Persistent expression of transfected genes. [49]
Jarraya et al. (2009) LV-induced bilateral putaminal expression of TH, GCH and AADC. MPTP-treated macaques (n = 26). Correction of motor deficits at 44 months. No observed dyskinesias. [50]
Palfi et al. (2014) Open label study. LV-induced putaminal expression of TH, GCH and AADC. Patients with advanced bilateral PD (n = 15). Therapy is deemed safe and tolerable. Improved UDPRS scores and lower need for medication. [51]

Clinical and preclinical studies with combined TH-, AADC- and GCH-gene therapy.