July 29, 2009
Xiao Xiao, PhD, the Fred Eshelman Distinguished Professor of Gene Therapy in the Division of Molecular Pharmaceutics, has received a five-year grant worth up to $1.66 million from the National Institute of Arthritis and Muscoskeletal and Skin Diseases of the NIH to support his research into treatments for Duchenne muscular dystrophy.
The grant will support Xiao’s project, “Myostatin Inhibition in DMD Dogs by Gene Transfer.” Muscular dystrophies are genetic diseases characterized by progressive muscle wasting. Duchenne muscular dystrophy occurs when a genetic mutation prevents the production of dystrophin, an essential muscle protein. Without this protein, individuals with DMD experience progressive loss of muscle strength and usually die in their twenties from respiratory or cardiac failure. There is currently no effective treatment for DMD, which affects approximately one out of every 3,500 boys worldwide.
In this NIH funded project, Xiao’s team will use a new approach to promote muscle growth and curb muscle wasting by means of gene therapy. They will deliver a therapeutic gene that blocks the action of the protein myostatin. Myostatin is produced by skeletal muscles to limit their growth and size. By blocking myostatin, normal muscles as well as dystrophic muscles will grow larger and stronger, Xiao says.
Xiao will deliver the myostatin-blocker gene to DMD dogs who have similar clinical symptoms as seen in the DMD patients in hope that muscle wasting will be reversed or halted.
“If this strategy works for DMD, it may also work for other forms of muscle-wasting diseases,” he says.
Xiao is collaborating on the project with with Joe Kornegay, DVM, PhD, in the UNC School of Medicine.
Xiao’s research group is also working to genetically engineer a nonpathogenic and defective DNA virus, named adeno-associate virus. They remove the original genes from the AAV and replace them with other genes, turning the virus into a twenty-two-nanometer delivery truck that ferries therapeutic genes to a variety of cells, tissues, and even the whole body. In addition to being efficienct, AAV has also proven to be very safe. Xiao has developed AAV-based treatments for diseases such as muscular dystrophies, heart failure, diabetes, arthritis, hepatitis, and cancer. A first-of-its-kind gene therapy for Duchenne muscular dystrophy is in a phase I clinical trial.
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