University researchers use stem cells to treat muscular dystrophy

Kali Dingman

University of Minnesota’s Lillehei Heart Institute found a way to effectively treat muscular dystrophy in mice by using human stem cells. This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

The publication, published today, shows how the dividing population of skeletal myogenic progenitor cells, or muscle-forming cells, derives from induced pluripotent cells. Induced pluripotent cells are patient-specific, which means they are less likely to be rejected and don’t involve destroying embryos.

University researchers were the first to use embryonic stem cells from mice to treat muscular dystrophy.

University researchers genetically modified two human iPS cell lines and an existing human ES cell line with the PAX7 gene.  This regulated levels of the Pax7 protein, which is vital for regenerating skeletal muscle tissue after damage and allowed naïve ES and iPS cells to differentiate into muscle-forming cells.

Researchers transplanted skeletal mygoenic progenitor cells into mice suffering from muscular dystrophy. The results showed an extensive and long-term muscle regeneration that resulted in improved muscle function.

Pax7-induced muscle progenitors were proven to be more effective than human myoblasts, previously tested for in clinical trials for muscular dystrophy, at improving muscle function.

The University researchers say more studies will need to be done before they can begin clinical trials.