A CRISPR possibility for DMD

CRISPR-based genome editing has filled the headlines since its discovery about two years ago for its ability to modify specific sequences in the genome with high accuracy, but its impact to date has been mainly as a research tool. A team from The University of Texas Southwestern Medical Center has now shown that the technology could have a therapeutic use in Duchenne muscular dystrophy, a disease involving mutations in the dystrophin gene.¹

CRISPR (clustered, regularly interspaced short palindromic repeats) and DMD might be an obvious marriage of technology and disease because there is a direct link between resolving the mutations and improving symptoms in DMD. But the technology faces an uphill path to solve the key challenge of how to scale and deliver it in humans...