Gene editing to reverse myotonic dystrophy

Using CRISPR in mice to reverse symptoms of the most common form of adult onset muscular dystrophy
22 September 2020
Presented by Chris Smith
Production by Eva Higginbotham.


Gene Editing CRISPR


Myotonic dystrophy type 1 is a debilitating disease that causes muscle weakness and wasting, eyesight problems, and heart defects, and there’s currently no cure. It’s a genetic condition linked to a gene called DMPK; this causes a toxic form of the chemical RNA - a genetic messenger molecule similar to DNA - to build up and compromise affected cells. They do this because the RNA molecules are sticky and they clog up other important processes inside cells. Now, scientists at the University of California San Diego have successfully used gene editing to reverse the disease in mice, as Eva Higginbotham heard from Gene Yeo...


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