Muscle fibers treated with a new RNA-targeted Cas9 technique show a lack of toxic RNA buildup (seen in red)

We've seen the powerful ways the CRISPR gene-editing tool could bring about better health outcomes by targeting and replacing specific pieces of DNA, but scientists at the University of California San Diego (UCSD) have been exploring a different approach. By using the technique to target RNA instead, the researchers have demonstrated an ability to destroy toxic accumulations of this molecule, which could pave the way for new treatments for muscular dystrophy and related conditions.

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Category: Medical, Science

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