Tuesday, May 26, 2009

New Therapy Substitutes Missing Protein in Those with Muscular Dystrophy

Source: University of Minnesota
Date: May 26, 2009

Summary:

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. In the mouse model, researchers were able to substitute for the missing protein – dystrophin, which forms a key part of the framework that holds muscle tissue together – that results in the disease, effectively repairing weakened muscle tissue.

Researchers injected dystrophic mice with a protein called utrophin – a very close relative of dystrophin – that was modified with a cell-penetrating tag, called TAT. The study is the first to establish the efficacy and feasibility of the TAT-utrophin-based protein as a viable therapy for the treatment of muscular dystrophy as well as cardiac muscle diseases caused by loss of dystrophin. The research is published in the May 26, 2009 issue of PLoS Medicine.