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article imageNanoparticles treat muscular dystrophy

By Tim Sandle     Feb 17, 2014 in Science
Researchers have demonstrated a new approach to treating muscular dystrophy. In studies using mice, the rodents showed improved strength and heart function when treated with nanoparticles.
For the research, the mice were conditioned to have a form of the muscle-weakening disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste. The nanoparticle used in the study consists of an inert core made of a chemical called perfluorocarbon, originally designed as a blood substitute during the 1960's.
Muscular dystrophy (MD) is a group of muscle diseases that weaken the musculoskeletal system and hamper locomotion. Muscular dystrophies are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue.
The faulty gene that causes the disease prevents the body from producing dystrophin, according to the research note. This is a protein crucial for maintaining muscle cell integrity and function. The new study demonstrated that mice with muscular dystrophy, in addition to missing dystrophin, also cannot recycle cellular waste, a process known as autophagy, or self-eating. The study showed that boosting autophagy improved skeletal muscle strength and heart function in these mice.
When treated with rapamycin nanoparticles, the mice showed a 30 percent increase in grip strength and a significant improvement in cardiac function, based on an increase in the volume of blood the heart pumped.
The research was undertaken at the Washington University School of Medicine in St. Louis. The results have been published in The FASEB Journal. The paper is titled “Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function”.
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