“Two and a half years ago, a study published in Science Advances detailed how the gene editing tool CRISPR/Cas-9 repaired genetic mutations related to Duchenne Muscular Dystrophy (DMD). The study was a proof of concept, and used induced pluripotent stem cells (iPSCs).
But now a similar treatment has not only been administered to real people, it has worked and made a difference in their quality of life and the progression of their disorder. Nine boys aged 6 to 12 who have been living with DMD since birth received a gene therapy treatment from pharmaceutical giant Pfizer, and a year later, 7 of the boys show significant improvement in muscle strength and function.
Though the treatment’s positive results are limited to a small group, they’re an important breakthrough for gene therapy, and encouraging not just for muscular dystrophy but for many other genetic diseases that could soon see similar treatments developed.”