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Science Magazine | AAAS: Science Translational Medicine

Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model | Science Translational Medicine

AAV9-mediated systemic delivery of a protein that targets CUG repeat RNA ameliorates myotonia in a mouse model of myotonic dystrophy.
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Image for the article: Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model | Science Translational Medicine
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