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First Parkinson gene therapy trial launches

Nature Biotechnology volume 21, pages 1117–1118 (2003)Cite this article

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The first gene therapy trial for Parkinson disease performed this August at New York Weill Cornell Medical Center (New York, NY, USA) joins a short list of other phase 1 clinical gene therapy trials aimed at brain disorders currently underway in the United States. Although the trial is groundbreaking, investors remain wary of safety concerns that loom over gene therapy—both in the brain and elsewhere.

The approach used by the researchers at Cornell, with technology rights owned by the biotech company Neurologix (Fort Lee, NJ, USA), is a departure from strategies established by consensus at workshops in 2000 held by the National Institute of Neurological Disorders and Stroke (NINDS; Bethesda, MD, USA). At these meetings, and in ongoing work by a Parkinson disease gene therapy consortium based at the University of Rochester (Rochester, NY, USA), scientists focus on either inserting genes into the brain to regulate the levels of dopamine or boosting growth factors to enhance survival of neurons, according to Diane Murphy, program director for neurodegeneration at NINDS. Cornell researchers are instead attempting to dampen the hyperkinetic movement characteristic of the disease by increasing production of γ-aminobutyric acid, a molecule that inhibits nerve activity.

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    Ken Howard

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Howard, K. First Parkinson gene therapy trial launches. Nat Biotechnol 21, 1117–1118 (2003). https://doi.org/10.1038/nbt879

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