Safer, More Efficient CRISPR Tech Offers Possible Duchenne Muscular Dystrophy Treatment

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We’ve written about CRISPR technology before, and it’s potential for treating sickle cell anemia. But in a latest study done at the University of California, Berkeley, sponsored by the NIH, the results could have a tremendous impact in the way of Duchenne Muscular Dystrophy (DMD) Treatment.

There has been hope that the CRISPR-Cas9 gene editing technology being developed could cure genetic diseases. It enables the editing of certain parts of the DNA sequence, allowing for precision and simplicity in an otherwise incredibly complex process. The challenge has been the method of delivery. One method is using viruses to deliver the gene editing, but that comes with complications.

UC Berkeley scientists have come up with a new way of delivering CRISPR-Cas9, called CRISPR-Gold, that doesn’t use viruses and they’ve now seen success in initial testing in mice with DMD.

In short, mice with DMD had a correction rate 18 times higher than control groups treated with Cas9 and donor DNA alone. Additionally, there scored double in strength and agility tests. This success means that there is a safer, more effective way to deliver CRISPR.

This new method of delivery actually corrected the DMD-causing genetic mutation and came with very little negative affects on DNA not targeted by CRISPR. The better news: This new method could be deployed for other genetic diseases as well. Offering hope to many in the rare disease community.

Human trials are the next step to determine if this technology is as safe and effective as seen in mice. To read the full release from UC Berkeley, click here.


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