Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial. (9th August 2022)

• Record Type: Journal Article
• Authors: Christine, Chadwick W.; Richardson, R. Mark; Fine, Elisabeth M.; Khwaja, Omar S.; Liang, Grace S.; Meier, Andreas; Roberts, Eiry W.; Bankiewicz, Krystof; Larson, Paul S.
• Is Part Of: Neurology. Volume 99:Number 6(2022)
• Page Start: 259
• Page End: 259
• Publication Date: 2022-08-09
• Subjects: Neurology -- Periodicals; Neurology -- Periodicals; Neurologie -- Périodiques; 616.8
• DOI: 10.1212/WNL.0000000000201003
• Languages: English
• ISSNs: 0028-3878

Abstract

Abstract : We appreciate the comment by Kang et al. on our research. 1 While we acknowledge findings of toxicity in a mouse model of elevated cellular dopamine due to overexpression of the dopamine transporter, 2 such toxicity has not been observed in rodent or primate models of Parkinson disease (PD) in which the aromatic L-amino acid decarboxylase (AADC) gene therapy was tested. 3, 4 Moreover, in our observations of these participants 3 years after AADC therapy, 1 we did not see evidence of significant clinical worsening, which would be expected if the AADC gene therapy was causing injury to medium spiny neurons.