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New CRISPR Method Takes on Duchenne Muscular Dystrophy

The advance of CRISPR gene editing technology, which uses an RNA strand to guide an enzyme called Cas9 to cut a specific portion of DNA, has raised concerns and sparked debate as people envision a not-so-distant future populated by bioengineered super-crops, genetically flawless pets, and customized babies. While the method could be used for these purposes, it’s also showing potential as a valuable medical tool, with a seemingly new condition added each week to the list of what CRISPR may one day cure.

One recent addition to that list is Duchenne muscular dystrophy (DMD). In a study from University of Texas Southwestern Medical Center, researchers used CRISPR to make a single cut at a few strategic points along DNA in cells derived from DMD patients, with the result of potentially correcting most of the 3,000 gene mutations that cause DMD.


New CRISPR Method Takes On Duchenne Muscular Dystrophy