CRISPR Gene Editing Cures Muscular Dystrophy in Mice

The CRISPR-Cas9 system allows genes to be edited easily and accurately, compared with previous gene-editing techniques (NEJM JW Gen Med Jun 15 2015 and Nature 2015; 520:186). Thousands of laboratories are using the technique to edit genes within cells in vitro.

Nevertheless, it is one thing to edit genes inside cells in a laboratory dish, but it's quite another to edit genes in the cells of particular diseased organs, deep within the body of a living animal. This complication has led some skeptics to predict that the CRISPR technique would never be used to treat human disease, although recent developments have improved the odds for successful targeting (NEJM JW Gen Med Mar 15 2016 and Nature 2016; 529:490).

Now, three different research group…