Journal
SCIENCE TRANSLATIONAL MEDICINE
Volume 1, Issue 2, Pages -Publisher
AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/scitranslmed.3000350
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The introduction of L-dopa (L-3,4-dihydroxyphenylalanine) therapy 40 years ago was a revolution in the treatment of patients with Parkinson's disease (PD). With time, however, the shortcomings of oral L-dopa medication became apparent, in particular the appearance of troublesome side effects, expressed as involuntary movements (dyskinesias) that developed over time in many patients. A gene therapy approach, aimed at restoring dopamine synthesis in the affected brain by viral vector delivery of genes that encode the dopamine-synthesizing enzymes, may off er a solution to this problem. Now, a team of French and UK researchers reports promising results in a nonhuman primate model of PD, paving the way for clinical trials of this enzyme-replacement approach.
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