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Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Yanlin Wang, Lei Hao, Hongcai Wang, Katherine Santostefano, Arjun Thapa, John Cleary, Hui Li, Xiuming Guo, Naohiro Terada, Tetsuo Ashizawa, Guangbin Xia

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Biochemistry, Genetics and Molecular Biology

Neuroscience