Research ArticleGene Therapy

Gene therapy reduces Parkinson’s disease symptoms by reorganizing functional brain connectivity

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Science Translational Medicine  28 Nov 2018:
Vol. 10, Issue 469, eaau0713
DOI: 10.1126/scitranslmed.aau0713

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The metabolic signature of gene therapy

Gene therapy delivering glutamic acid decarboxylase (GAD) is known to have therapeutic effects in patients with Parkinson’s disease (PD). However, the precise mechanisms mediating the improvements remain unclear. Now, Niethammer et al. used brain metabolic network analysis in patients with PD and showed that after gene therapy, patients developed a treatment-specific brain metabolic network involving motor-cortical regions. The network correlated with clinical outcome and was not affected by placebo effect. The results indicate that the therapeutic effects of GAD gene therapy are likely mediated by modulation of brain metabolism and suggest that metabolic network analysis might be useful for evaluating therapeutic efficacy in neurological disorders.