Skeletal muscle cells isolated using the ERBB3 and NGFR surface markers (right) restore human dystrophin (green) after transplantation significantly greater than previous methods (left).
UCLA researchers have developed ways to mature muscle cells in the laboratory to create muscle fibers that restore dystrophin, the protein missing in the muscles of boys with Duchenne Muscular Dystrophy, a muscle disease which affects approximately 1 in 5,000 boys in the U.S. and is the most common fatal childhood genetic disease. The findings are a major step towards developing a stem cell replacement therapy for Duchenne and other muscle diseases.
The study was published in the journal Nature Cell Biology by senior author April Pyle, associate professor of microbiology, immunology and molecular genetics and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. This research was supported by team science awards funded by the UCLA CTSI and the Center for Duchenne Muscular Dystrophy at UCLA.
For more information, see the press release from the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.