A new study led by Cincinnati Children's Hospital Medical Center finds muscular dystrophy may be able to be prevented. Jerry Lewis began raising money in the 1960s for the disease that decreases mobility and disrupts organ functions.
Children's researchers have tied the primary disease-causing component to part of the mitochondria, which processes nutrients into energy cells needed to survive. Scientists say if they prevent it from functioning, the disease vanishes in mice models.
"We have isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore," says the study's corresponding author Jeffery Molkentin, Ph.D., in a release. "If we prevent this pore from functioning, dystrophic disease in the mouse models we studied almost completely vanishes. We see the protection lasting past one year of life in the mouse, which translates to about 40 years of life for a human."
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Molkentin is a widely respected expert in the basic science of muscle cell function and formation. He is co-executive director of the Heart Institute at Cincinnati Children's and director of its Division of Cardiovascular Biology. He has studied muscular dystrophies for over 20 years.
Molkentin says more research is needed. He observed this discovery in genetically modified mice.
His research is published in the Aug. 25 issue of Science Advances.
An estimated 250,000 people in the U.S. are living with muscular dystrophy.
