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The therapeutic potential of gene transfer for the treatment of peripheral neuropathies

Published online by Cambridge University Press:  19 March 2007

James R. Goss
James R. Goss
Affiliation:
Molecular Genetics and Biochemistry, Center for Biotechnology and Bioengineering, University of Pittsburgh, 300 Technology Drive, Rm 208, Pittsburgh, PA 15219, USA. Tel: +1 412 383 9558; Fax: +1 412 383 9760; E-mail: jrgoss@pitt.edu
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Abstract

Peripheral neuropathy is a common medical problem with numerous aetiologies. Unfortunately, for the majority of cases there is no available medical solution for the underlying cause, and the only option is to try to treat the resulting symptoms. Treatment options exist when neuropathy results in positive symptoms such as pain, but there is a significant lack of treatments for negative symptoms such as numbness and weakness. Systemic application of growth factor peptides has shown promise in protecting nerves from neuropathic insults in preclinical animal studies, but translation into human trials has been problematic and disappointing. Significant advancements have been made in the past few years in utilising gene therapy approaches to treat peripheral neuropathy by expressing neuroprotective gene products either systemically or in specific nervous tissues. For example, plasmids expressing vascular endothelial growth factor injected into muscle, or herpes-simplex-virus-based vectors expressing neurotrophin gene products delivered to dorsal root ganglion neurons, have been used to protect peripheral nerve function in animal models of diabetes-associated peripheral neuropathy. Many published studies support the feasibility of this approach, although several questions still need to be addressed as gene therapy to treat peripheral neuropathy moves out of the laboratory and into the clinic.

Type
Research Article
Information
Expert Reviews in Molecular Medicine , Volume 9 , Issue 8 , March 2007 , pp. 1 - 20
Copyright
Copyright © Cambridge University Press 2007

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References

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Further reading, resources and contacts

The Neuropathy Association site provides general information and support services for patients suffering from peripheral neuropathy:

Poncelet, A.N. (1998) An algorithm for the evaluation of peripheral neuropathy. Am Fam Physician 57, 755-764 This is a useful reference in diagnosing and categorising various peripheral neuropathies.Google Scholar
Shy, M.E. (2006) Therapeutic strategies for the inherited neuropathies. Neuromolecular Med 8, 255-278. This excellent review discusses potential molecular targets for treating inherited neuropathies, including the use of gene therapy.Google Scholar
Davidson, B.L and Breakefield, X.O. (2003) Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4, 353-364.Google Scholar
http://www.neuropathy.org/site/PageServer Google Scholar
http://www.asgt.org/Google Scholar
Poncelet, A.N. (1998) An algorithm for the evaluation of peripheral neuropathy. Am Fam Physician 57, 755-764 This is a useful reference in diagnosing and categorising various peripheral neuropathies.Google Scholar
Shy, M.E. (2006) Therapeutic strategies for the inherited neuropathies. Neuromolecular Med 8, 255-278. This excellent review discusses potential molecular targets for treating inherited neuropathies, including the use of gene therapy.Google Scholar
Davidson, B.L and Breakefield, X.O. (2003) Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4, 353-364.Google Scholar
http://www.neuropathy.org/site/PageServer Google Scholar
http://www.asgt.org/Google Scholar