Abstract
Parkinson disease (PD) is a progressive neurodegenerative syndrome that affects motor and nonmotor abilities. This manufacturer-funded study addressed the safety, tolerability, and efficacy of bilateral intrastriatal delivery of ProSavin, a lentiviral vector–based gene therapy intended to enhance continuous dopamine production in the brain. The study was a phase 1/2 open-label trial with an open-label follow-up for safety. Fifteen patients with mild-to-moderate PD and a well-documented response to dopamine therapy received low-dose (1·9×107 transducing units [TU]; 3 patients), mid-dose …
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