University of California at Los Angeles (UCLA) researchers recently created skeletal muscle cells using human stem cells, marking a significant step toward perfecting stem cell replacement therapy for muscle diseases such as Duchenne muscular dystrophy.
The accomplishment followed years of efforts directed at duplicating functional muscle stem cells, according to a university release. Under the leadership of professor and stem cell researcher April Pyle, the UCLA team learned that existing methods were not satisfactorily efficient, generating immature cells.
“We have found that just because a skeletal muscle cell produced in the lab expresses muscle markers, doesn’t mean it is fully functional,” said Pyle. “For a stem cell therapy for Duchenne to move forward, we must have a better understanding of the cells we are generating from human pluripotent stem cells compared to the muscle stem cells found naturally in the human body and during the development process.”
The team identified a particular fetal skeletal muscle cell that they deemed “extraordinarily regenerative.” Discovering two cell surface markers, they isolated and grew the cells in the lab, producing muscle fibers, the release said. Subsequently, the scientists tweaked the cells to remove the mutation responsible for Duchenne.
Duchenne is a severe form muscular dystrophy affecting 1 out of every 5,000 boys nationwide, according to the release. Among childhood genetic diseases in the United States, it has the highest mortality rate.
The findings were published in Nature Cell Biology.