Gene Therapy and Stem Cell Technology Treat Rare Disease in Mice

Combining advances in gene therapy and stem cell technology, researchers report a potential treatment for a mouse model of Duchenne muscular dystrophy (DMD), a fatal genetic disease. Rita Perlgeiro with Michael Kyba and colleagues engineered mice with a mutation to the dystrophin and utrophin genes, much like human DMD counterparts. They then generated induced pluripotent stem (iPS) cell lines from mouse skin cells and employed a special gene-correcting tool called Sleeping Beauty Transposon to deliver the correct copy of the utrophin gene. Once integrated into the skin cells’ DNA, the researchers differentiated these cells into muscle cells for transplantation back into the DMD mice. Exceeding expectations, the iPS-derived muscle cells engrafted into disease sites and helped reduce symptoms of DMD.

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