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Genetic edits and protein restoration in mouse models of Duchenne muscular dystrophy remain viable one year after single CRISPR treatment
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Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes. The study appears online on February 18in the journal Nature Medicine.
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