New techniques in gene therapy improved muscle function in a murine model of muscular dystrophy, according to a recent study. Previous experiments in mice had been able only to prevent further muscle wasting.
The findings were published in the Sept. 16 online Early Edition of the Proceedings of the National Academy of Sciences.
In earlier work with newborn mice who lacked the dystrophin gene, researchers had been able to deliver the gene through adenovirus vectors. However, because the vectors had viral properties that provoked an immune response and because the dystrophin gene is large, results were limited.
In the Sept.16 paper, researchers reported the development of stripped-down vectors that did not raise an immune response. The vectors delivered the full-length dystrophin gene to the muscles of young and old mice, even those with severe muscle damage. Restoration of normal muscle function directly correlated to the amount of gene delivered. However, these results were only in specific small muscles, not throughout the body.
Co-authors of the paper included Christiana DelloRusso, Jeannine M. Scott, Dennis Hartigan-O’Connor, and Robert W. Crawford of the UW Department of Neurology, former UW researchers Giovanni Salvatori, Catherine Barjot and Ann S. Robinson, Susan V. Brooks of the University of Michigan, and Jeffrey S. Chamberlain, UW professor of neurology,
The National Institutes of Health, the Muscular Dystrophy Association, and the Apex Foundation, established by Bruce and Jolene McCaw, funded the research.
September 20, 2002