UCLA researchers have developed a potential stem cell based treatment for Duchenne Muscular Dystrophy (DMD). This degenerative muscular disorder - caused by a genetic mutation in a protein essential for skeletal muscle function - primarily affects young men and boys, degenerating skeletal muscle to the point where it eventually wastes away. This severe degeneration of skeletal muscle results in almost a complete lack of voluntary movement. The UCLA team used the gene editing technology, CRISPR, to repair the mutated gene for the protein in stem cells obtained from the subjects and reintroduced the re-programmed muscle cells back into the subjects. In an animal model, the re-programmed cells successfully produced the dystrophin protein, reversing the degeneration of the skeletal muscle. The team has also conducted a second set of biological markers to ensure complete differentiation into adult muscle cells.
Researchers led by Eduardo Marbón of the Cedars-Sinai Heart Institute have developed a method to prolong the lives of patients with Duchenne Muscular Dystrophy [DMD] through the infusion of cardiac stem cells. The stem cells reverse the loss of cardiac muscle caused by the genetic disease, preventing heart failure that would otherwise limit a patient’s life expectancy to age 25.
Researchers at Sanford-Burnham Medical research institute have developed a potential method of regenerating autologous [the patient’s own] stem cells to repair damaged muscle tissue in patients suffering from muscle-degenerative diseases. The scientists found that the inhibition of protein STAT3 results in the replenishment of the body’s muscle stem cells, which in turn repair the muscles that are damaged by age, cancer, and diseases such as muscular dystrophy.
In a recently published study by the University of Minnesota, researchers are utilizing skeletal muscle stem cells in an animal model designed to study the muscle-degenerative protein DUX4 found in patients with facioscapulohumeral muscular dystrophy [FSHD]. The team was able to harvest the muscle stem cells from one mouse with FSHD and transplant them into a recipient mouse, allowing the recipient to regenerate new muscle as long as DUX4 was not activated.
A recently published study by University of Illinois Kinesiology and Community Health Professor Marni Boppart has identified mesenchymal stem cells [MSCs] as a tool for rejuvenating muscle to prevent age-related injuries and disabilities. In addition to their ability to differentiate into other cell types, MSCs were found to secrete growth factors that stimulate the activation of the multiple cell types comprising skeletal muscle, including muscle precursor cells and satellite cells, which lose function with age.
Recent research from the University of Colorado Boulder has identified two stem cell signaling pathways that are directly related to the age-related degeneration of muscle tissue and the onset of a variety of muscle-wasting diseases, such as muscular dystrophy and sarcopenia. By altering these pathways, the investigators were able to enhance muscle stem cell renewal and improve muscle regeneration.
Duchenne Muscular Dystrophy, a particularly severe form of muscular dystrophy [MD - a genetic disorder], affects as many as 15,000 young Americans. Many of those afflicted eventually succumb to cardiac or respiratory failure by their early 20’s. Researchers at University of California, Los Angeles, are utilizing stem cells to model the disease in order to develop and test treatments. One particularly promising treatment involves a method referred to as exon skipping. The technique essentially tricks the cell's machinery into misreading the MD genetic mutation so instead of producing the defective protein responsible for the disease, the cells produce a more functional version of the protein. In using muscle cells derived from stem cells of people with muscular dystrophy – the cells contain the mutation that causes muscular dystrophy - researchers anticipate their tests to more accurately reflect how human cells would react to their drugs [or combination of drugs]. This enables more efficient and effective testing of potential treatments and speeds the process of developing those treatment options that show the most promise. According to Dr. Stanley Nelson, a lead researcher on the project, “We are thrilled that stem cell research will change the outcome of Duchenne”.