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.