Gene therapy for dopamine replacement in Parkinson's disease.

Research output: Contribution to journalArticle

Abstract

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 offer 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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Subject classification (UKÄ) – MANDATORY

  • Neurosciences
Original languageEnglish
Pages (from-to)2ps2
JournalScience Translational Medicine
Volume1
Issue number2
Publication statusPublished - 2009
Publication categoryResearch
Peer-reviewedYes