«This work demonstrates the feasibility
of using a
single gene editing platform, plus the regenerative
power of stem cells to correct genetic mutations and restore dystrophin production for 60 percent
of Duchenne patients,» said Pyle, associate professor
of microbiology, immunology and molecular genetics and member
of the Broad Stem Cell Research Center.
In a sign
of the method's
power, Pritchard's team also detected selection in traits controlled not by a
single gene, but by tiny changes in hundreds
of genes.