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.
Dr. Pyle, senior author of the study and Associate Professor at the Center of Regenerative Medicine and Stem Cell Research at UCLA comments, “The results were exactly what we’d hoped. This is the first study to demonstrate that functional muscle cells can be created in a laboratory and restore dystrophin in animal models of Duchenne using the human development process as a guide.”
Advances in stem cell based regenerative treatments are creating more effective treatment options for patients suffering from a wide variety of disease, trauma and injury. To learn more about stem cells, and how families can bank their own valuable stem cells by recovering the very powerful dental pulp stem cells during routine dental procedures; such as wisdom teeth extractions or the during the loss of baby teeth, visit StemSave or call 877-783-6728 (877-StemSave) today.
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