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Research Shows Promise for New Muscular Dystrophy Treatments

Posted on July 27, 2018


A new process developed by researchers at the University of Minnesota Medical School may mean a new treatment for individuals living with muscular dystrophy.

The project uses cells from an unusual source: a tumor known as a teratoma.

Teratomas are tumors made up of tissues such as hair, muscle, teeth and bone. They often occur in the gonads – in the ovaries in women or in the testicles in men.

Teratomas are typically benign, but can be malignant, or cancer-causing, in some cases.

The study, published in the journal Cell Stem Cell, was conducted by the Lillehei Heart Institute. The goal of the research was to find sources of stem cells to help rebuild skeletal muscle and increase muscle strength and flexibility.

The researchers focused on teratomas found in animals and discovered that they could refine and sort the cells of the tumor and use these cells to recreate skeletal muscle in mice affected by muscular dystrophy.

The teratomas researchers used were not those that occurred naturally in the mice. Instead, the study authors created teratomas using injected undifferentiated pluripotent stem cells. These teratomas contained many different types of cells, including muscle stem cells.

Next, the researchers took the muscle stem cells found in the grown teratomas and injected them into mice with muscle tissue affected by muscular dystrophy. They found that these cells regenerated 80 percent new muscle tissue – a significant increase over the 5 to 10 percent regeneration previous research has made possible.

The muscle stem cells that were taken from the teratomas also filled the new muscle tissue with muscle stem cells.

The new muscle tissue also proved to be superior to other created muscles in terms of tetanic force, or sustained muscle contraction. The tissues also rated higher regarding specific force and fatigue time.

“The ability to repair muscle tissue is critical for the treatment of muscular dystrophy and other muscle conditions. It is also critical to ensure that the new muscle is strong,” said Dr. Joel Singer.

Singer is a New York physician who treats patients living with muscular dystrophy and other muscle-related diseases using personal cell therapy.

“This cell therapy can help individuals living with muscle conditions like muscular dystrophy,” said Singer.

Although the results are exciting and the ability to create new muscle tissue could have an incredible impact on treating muscular dystrophy and other illnesses, there is a long way to go before human trials.

The total number of people living with muscular dystrophy is hard to calculate because there are more than 30 genetic diseases that fall under the neuromuscular disease umbrella. Some of the conditions in this category include amyotrophic lateral sclerosis, myotonic dystrophy and spinal muscular atrophy.

These conditions are all characterized by genetic mutations that interrupt the growth of healthy muscle tissue.

According to the U.S. Centers for Disease Control and Prevention, fewer than 200,000 people are diagnosed with muscular dystrophy each year.

Muscular dystrophy causes pain, fatigue, muscle weakness and muscle loss.

During the onset of the disease, the extremities are usually impacted first. Numbness and weakness develop, making movement difficult. As the condition progresses, these symptoms can affect other body areas, such as the lungs, heart and throat.

Some forms of muscular dystrophy can be fatal.

Both men and women can develop muscular dystrophy, but some forms of the condition, like Duchenne muscular dystrophy, affect males more frequently. DMD affects one in every 1,500 men in the U.S.


University of Minnesota Medical School. “Muscle stem cells derived from teratomas.” ScienceDaily. ScienceDaily, 5 July 2018.



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