Viral vectors for neurotrophic factor delivery:
A gene therapy approach for neurodegenerative diseases of the CNS




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Seung Lima, Mikko Airavaarab and Brandon K. Harveyb, ,

aDepartment of Neuroscience, Georgetown University Medical Center, Washington, D.C, 20057, United States

bIntramural Research Program, National Institute on Drug Abuse, National Institutes of Health, Baltimore, MD 21224, United States


Received 18 June 2009; revised 11 October 2009; accepted 11 October 2009.
Available online 17 October 2009.
Abstract
The clinical manifestation of most diseases of the central nervous system results from neuronal dysfunction or loss. Diseases such a stroke, epilepsy and neurodegeneration (e.g. Alzheimer's disease and Parkinson's disease) share common cellular and molecular mechanisms (e.g. oxidative stress, endoplasmic reticulum stress, mitochondrial dysfunction) that contribute to the loss of neuronal function. Neurotrophic factors (NTFs) are secreted proteins that regulate multiple aspects of neuronal development including neuronal maintenance, survival, axonal growth and synaptic plasticity. These properties of NTFs make them likely candidates for preventing neurodegeneration and promoting neuroregeneration.

One approach to delivering NTFs to diseased neurons is through viral vector-mediated gene delivery. Viral vectors are now routinely used as tools for studying gene function as well as developing gene-based therapies for a variety of diseases. Currently, many clinical trials using viral vectors in the nervous system are underway or completed, and seven of these trials involve NTFs for neurodegeneration. In this review, we discuss viral vector-mediated gene transfer of NTFs to treat neurodegenerative diseases of the central nervous system.

Keywords: Neurotrophic factor; Neurodegeneration; Gene therapy; Viral vector; adeno-associated virus; aav; lentivirus; herpes virus; HSV; adenovirus; Parkinson's disease; Alzheimer's disease; amyotrophic lateral sclerosis; stroke; epilepsy; huntington's disease; central nervous system; transgene; clinical trial


Corresponding author. National Institute on Drug Abuse, National Institutes of Health, BRC Suite 200, Rm 06A729, 251 Bayview Blvd, Baltimore, MD 21224. Tel.: +1 443 740 2590

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