VS Duchenne: Editing DNA to Cure Disease
First, Gavriel’s muscles got weaker. Then, he lost the ability to walk. From there, things will only get worse. The cause is Duchenne Muscular Dystrophy, a genetic disorder with no cure. But that might soon change. Using CRISPR, a state-of-the-art ...
SickKids VS · SickKids Foundation
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Show Notes
First, Gavriel’s muscles got weaker. Then, he lost the ability to walk. From there, things will only get worse. The cause is Duchenne Muscular Dystrophy, a genetic disorder with no cure. But that might soon change. Using CRISPR, a state-of-the-art gene-editing tool, SickKids scientists were able to correct the disease-causing mutation in mice. The result? Improved muscle strength and function—and no signs of paralysis. Now, they’re refining their approach, so they can take this treatment out of the lab and into the world.For more information on this episode, visit sickkidsfoundation.com/podcast/duchenne. And to fund research like this, go to sickkidsfoundation.com/donateSickKids Foundation is proud to recognize CIBC as the Premier Sponsor of the SickKids VS Podcast.