Duchenne MD Therapy by Gene Editing of Stem Cells May Be Possible in Next Decade

UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…