CRISPR gene-editing tool tweaked to tackle muscular dystrophy via RNA – New Atlas

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


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.

The new research builds on a study published in 2016, in which UCSD scientists showed that the CRISPR tool could be used to target RNA in live cells. RNA acts as the messenger service, communicating information about our genes to the cell’s machinery for producing proteins, and where it is located and how it travels through the cell can determine how well this important function is carried out.

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