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Gene Transfer to the Nervous System: Prospects for Novel Treatments Directed at Diseases of the Aging Nervous System

¹ Department of Neurology
² Department of Molecular Genetics and Biochemistry, University of Pittsburgh, Pennsylvania.
³ Geriatric Research, Education and Clinical Center, Pittsburgh VA Healthcare System, Pennsylvania.

In the past 3 decades, gene therapy has moved from a theoretical construct to an active field of basic research, animal studies, and clinical trials. In this article, we describe the conceptual basis underlying the use of gene therapy for diseases of the aging nervous system, the principal techniques used for gene delivery, and review preclinical animal studies in 4 different classes of neurologic dysfunction: 1) focal neuronal degeneration in the central nervous system; 2) global neuronal dysfunction in the central nervous system; 3) degenerative disease affecting components of the peripheral nervous system; and 4) intractable focal pain. The full potential of this approach will not be established until the human trials are completed.