Gene therapy for neurologic disease
S. T. Suhr and F. H. Gage
Department of Neurosciences, University of California, San Diego.
Gene therapy is a potentially potent new method of treating a number of
neurologic disorders previously considered refractory to current
conventional therapeutic treatments. Numerous advances have been made in
the construction of expression vectors, cellular and viral transgene
carriers, and the characterization of target cells for neuronal gene
therapy. Two primary approaches to nervous system gene transfer have
emerged as a result of these advances. The in vivo approach concentrates on
direct transfer of genetic material to cells in vivo using viral and
chemical agents. The ex vivo approach relies on genetic transfer to
cultured cells that are subsequently implanted into a host organism. Both
of these methods have been used in preliminary experiments designed to test
the efficacy of gene transfer strategies in the amelioration of nervous
system dysfunction.
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