329Radiographic Safety of MRI-Guided AAV2-AADC Infusion in Children With AADC Deficiencydoi:10.1227/neu.0000000000003360_329Aromatic amino-decarboxylase (AADC) deficiency is a rare inherited enzymatic disorder inhibiting dopamine and serotonin production, leading to severe developmental delay, hypotonia, ...
Objective: We investigated the safety and efficacy of delivery of a viral vector expressing AADC (AAV2-hAADC) to the midbrain in children with AADC deficiency. Design, Setting and Participants: Seven (7) children, aged 4-9 years underwent convection-enhanced delivery (CED) of AAV2-hAADC to...
Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial. PLoS One. 2017 Feb 6; 12(2): e0169965. doi: 10.1371/journal. pone.0169965. eCollection 2017....