New therapy created for muscular dystrophy
U.S. medical scientists say they’ve developed a new therapy for Duchenne musclar dystrophy, a fatal disease and the most common form of MD in children.
Researchers at the University of Minnesota Medical School, using a mouse model, said they were able to substitute for the missing protein dystrophin, which forms a key part of the framework that holds muscle tissue together.
The scientists injected dystrophic mice with a protein called utrophin — a close relative of dystrophin — that was modified with a cell-penetrating tag, called TAT.
The researchers said their 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.
This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches, Professor James Ervasti, who led the research, said.
The findings appear in the journal PLoS Medicine.