Discovery made while editing genetic defect behind Duchenne muscular dystrophy Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases. The finding stemmed from research at UT Southwestern in which a single-cut gene-editing

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01 March 2019 P.M.Fornasari Rare diseases affect fewer than roughly five in 10,000 people worldwide. Approximately 80% of rare diseases have identified genetic origins, and are often debilitating; about 30% of children with a rare disease will die before their fifth birthday. There are roughly 30 million people in the U.S. living with a rare

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