Magnetic resonance imaging–guided phase 1 trial of putaminal <i>AADC</i> gene therapy for Parkinson's disease

Abstract

Novel intraoperative monitoring of administration facilitated targeted delivery of VY-AADC01 in this phase 1 study, which was well tolerated. Increases in enzyme expression and clinical improvements were dose dependent. ClinicalTrials.gov Identifier: NCT01973543 Ann Neurol 2019;85:704-714.