Mayo reports on early preclinical results with interfering RNA for Parkinson's

A group at the Mayo clinic have reported preclinical results of a new approach to treating Parkinson's disease.
Previous studies found that variations in the alpha-synuclein gene result in increased protein production and are sufficient to cause Parkinson’s disease in some families. The gene produces a protein that accumulates at the site and is linked with progress of the disease.
The Mayo team designed small RNA molecules which would interfere with process by which the alpha-synuclein protein is produced. Infusion of the small interfering RNA into the brains of mice effectively reduced alpha-synuclein production for up to three weeks with no ill-effects on the mice.
Work by other teams seeking to use gene therapy techniques to reprogram brain cells to continuously produce sufficient interfering RNAs to suppress target gene activity may offer a route to potential clinical treatment.
The publication is available here.

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