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Exp Neurol. 1997 Mar;144(1):74-81.

Targets for gene therapy of Parkinson's disease: growth factors, signal transduction, and promoters.

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Department of Neurosurgery, Yale Univeristy School of Medcine, New Haven, Connecticut 06520, USA.


Parkinson's disease gene therapy is in its infancy. All studies to date have been in experimental animals and there are no clinical protocols currently approved. Several non-human primate studies however, have been completed and preliminary data appear promising. When dealing with a complex acquired disorder of unknown etiology, gene therapy is likely to provide symptomatic and palliative relief at best and will not be curative. However, if the gene therapy approach has advantages in terms of the risk/benefit ratio, cost and efficacy over current treatments, then it should be brought to clinical trial. This article discusses some future directions and areas of intense investigation at present. The advances in the field over the past five years have been tremendous and it appears possible that before the year 2000, clinical gene therapy trials in Parkinson's disease will be ongoing.

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