NEW YORK (Reuters Health) - Intravenous administration of adeno-associated viral vectors allows delivery of genes to the heart and skeletal muscles of mice, researchers report in a July 25th advanced online publication of Nature Medicine. This might be of use in therapy of conditions such as muscular dystrophy, they suggest.
“Our work represents a proof of principle that it is possible to deliver new genes to all the muscles of an adult animal,” senior investigator Dr. Jeffrey S. Chamberlain told Reuters Health. “The immediate goal is to find out if we can extend this work to people.”
Dr. Chamberlain and colleagues at the University of Washington in Seattle note that a systemic approach to the procedure is hampered by the vascular epithelium, which is “a barrier to distribution of vectors via the circulation.”
However, by using an adeno-associated virus along with vascular endothelial growth factor (VEGF) the barrier was penetrated in an animal model. In particular, this allowed widespread muscle-specific expression of functional micro-dystrophin in the skeletal muscles of dystrophin-deficient mice, a Duchenne dystrophy model.
“Additional studies,” Dr. Chamberlain, continued, “are being carried out to explore in detail the safety of the method.”
Should this particular approach ultimately prove safe and effective in humans, he concluded, “we hope that this same method could be used to deliver a gene for any type of muscular dystrophy, or even other diseases of voluntary muscles, including the heart.”
Nat Med, July 25, 2004 Advanced Online Publication.
MeSH Headings: Animal Diseases : Biological Therapy : Contractile Proteins : Cytoskeletal Proteins : Genetic Engineering : Genetic Techniques : Membrane Proteins : Investigative Techniques : Muscle Proteins : Muscular Dystrophy, Animal : Therapeutics : Gene Therapy : Dystrophin : Analytical, Diagnostic and Therapeutic Techniques and Equipment : Diseases
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