ScienceDaily (May 27, 2009) — Researchers at the University of Minnesota Medical School have discovered a new therapy that shows potential to treat people with Duchenne muscular dystrophy, a fatal disease and the most common form of muscular dystrophy in children.
"This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches," said James Ervasti, Ph.D., principal investigator of the study and a professor in the Department of Biochemistry, Molecular Biology & Biophysics.
What Do These Findings Mean?
These findings provide the first demonstration that injection of TAT-utrophin protein fusions may provide a way to treat muscular dystrophies caused by the loss of dystrophin. However, although this direct protein-replacement therapy looks hopeful, approaches that work in animals do not necessarily work in people. In particular, for this approach to work in patients with muscular dystrophy, it would be necessary to give frequent, high-dose injections of the TAT-μUtr fusion protein, a process that could eventually trigger a deleterious immune response. Nevertheless, the researchers suggest that by combining this novel approach with other approaches that also increase utrophin expression, it might be possible to prevent or delay the development of the symptoms of Duchenne muscular dystrophy.
article link: http://www.sciencedaily.com/releases/2009/05/090526152713.htm
research report link: http://www.plosmedicine.org/article/info%3Adoi%2F10.1371%2Fjournal....