UF researcher awarded grant to develop muscular dystrophy treatment

The Muscular Dystrophy Association has awarded a UF gene therapy researcher $2.2 million to develop a genetic treatment for muscular dystrophy that can be tested in people.

Barry Byrne, M.D, Ph.D., co-director of UF’s Powell Gene Therapy Center, a part of the UF Genetics Institute, was awarded the three-year grant to establish the safety of two different types of a gene therapy vector that can be used to deliver genes to muscle tissue to correct the defect that causes the progressive muscle breakdown associated with the disease. Byrne also is an associate professor in the UF College of Medicine’s departments of pediatrics, and molecular genetics and microbiology.

In conjunction with researchers Kevin Campbell, Ph.D., of the University of Iowa and Jerry Mendell, M.D., at the Ohio State University, Byrne will attempt to establish the long-term safety of the gene therapy vectors AAV1 and AAV2 in mice, dogs and primates with muscular dystrophy. Once their safety is established in animals, the vectors can be used in human studies, possibly within two years.

Muscular Dystrophy is a group of genetic diseases that cause progressive degeneration of the skeletal muscles. Most forms are caused by a defective gene, known as dystrophin. In Duchenne muscular dystrophy, the most common form affecting children, death usually occurs by the early 20s. Research has provided a better understanding of these disorders, but no treatments have been developed.

The most common and best understood vector now used to transport corrective genes in muscle is a specific type of the adeno-associated virus called AAV2.

But recently, another form of the virus, called AAV1, has been shown to be 100 to 1,000 times more efficient in transferring corrective genes to rodent muscle tissue. Unlike its more common cousin, AAV1 has never been used in clinical studies, and researchers have yet to determine the efficacy and safety in humans. Recognizing its potential, however, Byrne will study the vector more closely to determine its safety and effectiveness.

The vectors, which must be modified to carry corrective genes for each different type of genetic disease, will be manufactured at UF’s Human Applications Laboratory and Vector Core at the Evelyn F. and William L. McKnight Brain Institute of UF.