Scientists Reprogram Stem Cells to Regenerate Muscle in Muscular Dystrophy


Source: Northwestern University

Summary: In a new study, researchers have reprogrammed stem cells and demonstrated the potential to regenerate lost muscle mass in muscular dystrophy.


Muscular dystrophy is a group of inherited muscle diseases characterized by the progressive loss of both skeletal and cardiac muscle mass. The most common type muscular dystrophies include Duchenne muscular dystrophy, Becker muscular dystrophy, facioscapulohumeral muscular dystrophy, and myotonic dystrophy. While there is no proper cure but some physical therapies and medication can offer relief for symptoms, and recent advancements in induced pluripotent stem cells (iPSCs) have pointed to the possibility of future muscle regeneration therapies. In a new study, researchers from the Northwestern University have reprogrammed stem cells and demonstrated the potential to regenerate lost muscle mass in muscular dystrophy. The research findings were published in the journal Nature Communications.

reprogrammed stem cells regenerate lost muscle mass in muscular dystrophy.

Images showing MiPs inducing regeneration of both heart and muscle tissue (green), and subsequent destruction of regenerated tissue when the MiPs are turned off by the molecule AP20187. Credit: Northwestern University

Past research have shown that mouse-derived mesodermal iPSC-derived progenitor (MiP) stem cells can spur muscle regeneration in mice. To check if human stem cells can regenerate lost muscle tissue, the researchers injected human MiP cells into mouse models and found increased heart volume and improved muscle structure compared to controls. When they later introduced a drug to downregulate the MiPs, the beneficial effects were reversed, supporting the evidence that human MiPs have regeneration potential. When MiP cells created from skeletal muscle mesoangioblasts (MAB) were used it resulted in the generation of more skeletal muscle cells when compared to MiPs derived from fibroblasts. But treating fibroblast MiPs with microRNA cocktails showed even more promise, greatly improving the skeletal muscle differentiation of fibroblast MiPs.

Mattia Quatrocelli said, “One of the most innovative aspects of this study is the identification of actionable molecules—the microRNA cocktails—to improve the innate efficiency of functional amelioration that human MiPs can impart on dystrophic muscle”, “The next step will be to capitalize on these discoveries by improving safety and bringing this novel therapeutic option closer to clinical standards.”


More Information: Giorgia Giacomazzi et al, “MicroRNAs promote skeletal muscle differentiation of mesodermal iPSC-derived progenitors”, Nature Communications(2017). DOI: 10.1038/s41467-017-01359-w


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