CRISPR genome editing corrects a Duchenne muscular dystrophy deletion mutation in mice
Appeared in BioNews 1093
Two genome editing approaches can correct a variety of mutations associated with Duchenne muscular dystrophy (DMD), researchers have shown.
DMD is a genetic disorder caused by mutations in the
dystrophingene, leading to a fatal decline in muscle integrity. For the first time, researchers have used two CRISPR-based genome editing techniques to correct multiple dystrophin mutations in mice. It is hoped this could pave the way for new genome editing therapies for DMD.
'Every cell in the human body has three billion letters of DNA sequence in its genome, and this method makes it possible to correct large deletions in the DMD gene by specifically swapping one of these letters,' said Professor Eric Olson, of the University of Texas Southwestern Medical Centre in Dallas, and senior author of the study. 'That level of specificity and efficiency is remarkable,' he added.