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Prospects for gene therapy in Parkinson's disease
Author(s) -
Freese Andrew,
Stern Matthew,
Kaplitt Michael G.,
O'Connor William M.,
Abbey Maureen V.,
O'Connor Michael J.,
During Matthew J.
Publication year - 1996
Publication title -
movement disorders
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.352
H-Index - 198
eISSN - 1531-8257
pISSN - 0885-3185
DOI - 10.1002/mds.870110502
Subject(s) - genetic enhancement , parkinson's disease , ex vivo , clinical trial , disease , neuroscience , medicine , degenerative disease , bioinformatics , in vivo , gene , biology , pathology , genetics
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 genes 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.