Gene Therapy in a Nonhuman Primate Model of Parkinson's Disease

摘要

In the final year of this grant, we are completgeing an experiment in which aged monkeys are assessed for levodopa-induced dyskinesias and given a fluorodopa PET scan. They then receive lenti-ODNF under the control of the tetracycline promoter. Three months following lenti-ODNF, they are again assessed for levodopa-induced dyskinesias and given a fluorodopa PET scan. Then half of the aged monkeys receive tetracycline to shut of the GDNF expression in vivo. Monkeys are assessed for levodopa-induced dyskinesias and given a fluorodopa PET scan 3 and 6 months later. This study will determine whether we can control gene expression in vivo and make gene therapy safe for clinical use.