Prospects for gene therapy in Parkinson's disease.

Publication Type Review
Authors Freese A, Stern M, Kaplitt M, O'Connor W, Abbey M, O'Connor M, During M
Journal Mov Disord
Volume 11
Issue 5
Pagination 469-88
Date Published 09/01/1996
ISSN 0885-3185
Keywords Genetic Therapy, Parkinson Disease
Abstract Numerous advances in in vivo and ex vivo gene-therapy approaches to Parkinson's disease offer promise for direct clinical trials in patients in the next several years. These systems are predicated on introducing gene that encode enzymes responsible for dopamine biosynthesis or neurotrophic factors that may delay nigrostriatal degeneration or facilitate regeneration. We review the current status of experimental approaches to gene therapy for Parkinson's disease. Comparative advantages and disadvantages of each system are enumerated, and preclinical trials of some of the systems are evaluated. Although the specific in vivo or ex vivo methods used for gene transfer into the brain are likely to be supplanted by newer technology over the next decade, the principles and approaches developed in current studies likely will remain the same.
DOI 10.1002/mds.870110502
PubMed ID 8866488
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