BioCentury
ARTICLE | Targets & Mechanisms

Snipping vs. skipping for DMD

Why CRISPR might be better than exon skipping for DMD

January 21, 2016 8:00 AM UTC

As if to preempt the regulatory setbacks in Duchenne muscular dystrophy (DMD) that last week disappointed the field, a trio of preclinical studies emerged two weeks earlier showing that cutting out DMD mutations with gene editing might offer a viable alternative to the exon-skipping strategies that have dominated the pipeline. Now, the question is whether there's reason to believe the mouse studies will translate any better to the clinic.

The studies, published Dec. 31 in Science, provide in vivoproof of concept for the first time that CRISPR-Cas9 used postnatally can have a disease-modifying effect. Despite the hype around its therapeutic promise, the technology has so far proved itself primarily in research applications, for example, in modifying cells for in vitroscreening or creating animal models of disease...