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Local and global gene therapy in the central nervous system

Published online by Cambridge University Press:  04 February 2010

Leslie L. Muldoon  and
Edward A. Neuwelt
Leslie L. Muldoon
Affiliation:
Department of Cell Biology and Anatomy;
Edward A. Neuwelt
Affiliation:
Department of Neurology, Division of Neurosurgery Department of Biochemistry and Molecular Biology, Oregon Health Sciences University, Portland, OR 97201. [email protected]
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Abstract

For focal neurodegenerative diseases or brain tumors, localized delivery of protein or genetic vectors may be sufficient to alleviate symptoms, halt disease progression, or even cure the disease. One may circumvent the limitation imposed by the blood-brain barrier by transplantation of genetically altered cell grafts or focal inoculation of virus or protein. However, permanent gene replacement therapy for diseases affecting the entire brain will require global delivery of genetic vectors. The neurotoxicity of currently available viral vectors and the transient nature of transgene expression in vivo must be overcome before their use in human gene therapy becomes clinically applicable.

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Authors' Responses
Information
Behavioral and Brain Sciences , Volume 18 , Issue 1: An International Journal of Current Research and Theory with Open Peer Commentary , March 1995 , pp. 76 - 78
Copyright
Copyright © Cambridge University Press 1995

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