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Gene Therapy for Neurologic Disease

Gene Therapy for Neurologic Disease Abstract 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. References 1. Gage FH, Wolff JA, Rosenberg MB, et al. 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Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters . Nature . 1993;361: 726-730.Crossref http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Archives of Neurology American Medical Association

Gene Therapy for Neurologic Disease

Suhr, Steven T.; Gage, Fred H.
Archives of Neurology , Volume 50 (11) – Nov 1, 1993
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References (166)

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American Medical Association
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Copyright © 1993 American Medical Association. All Rights Reserved.
ISSN
0003-9942
eISSN
1538-3687
DOI
10.1001/archneur.1993.00540110122012
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Abstract

Abstract 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. References 1. Gage FH, Wolff JA, Rosenberg MB, et al. 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Journal

Archives of Neurology – American Medical Association

Published: Nov 1, 1993

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