gene therapy for Parkinson's
A Yale neurosurgeon received a $5.5 million federal translational grant to develop two promising vector systems for delivery of gene therapy for Parkinson's disease.
Dr. D. Eugene Redmond, professor of psychiatry and neurosurgery at the School of Medicine, will test in primates the effectiveness and safety of the human growth factor GDNF (human glial cell line derived neurotrophic factor) delivered by two improved vector systems -- one derived from equine infectious anemia virus and the other from adenoassociated virus.
The vectors use the ability of the virus to get into cells and into the machinery of the cell. The part of the virus that causes it to reproduce is removed; the DNA of a gene is inserted in its place; and the newly configured vector then enters the cell and directs it to make something useful.
"The 'something useful,' in this case, is GDNF," Redmond says. "GDNF has shown promise for preventing or reversing morphological, biochemical and functional deficits in other models of Parkinson's disease in rodents and primates using similar viral vectors."
Redmond says his team's studies also showed important problems that need to be solved to ensure that GDNF gene therapy will be safe and effective in patients. Concerns about inflammation, toxicity, inadequate or excessive gene expression, persistence and viral recombination or replication have led to the development of improved and safer vectors with regulatable promoters, which will be tested in this proposed project.
The project will be carried out in collaboration with investigators from St. Kitts Biomedical Research Foundation, West Indies; the Swiss Federal Institute of Technology, Lausanne, Switzerland; and the University of North Carolina.
The funding from the National Institutes of Health (NIH) is a translational grant, which indicates that the NIH believes the research is on the verge of final studies and on the threshold of clinical trials involving humans by the end of the five-year grant period.
-- By Jacqueline Weaver
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