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    Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

    Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy by Audrey M. Winkelsas, Christopher Grunseich, George G. Harmison, Katarzyna Chwalenia, Carlo Rinaldi, Suzan M. Hammond, Kory Johnson, Melissa Bowerman, Sukrat Arya, Kevin Talbot, Matthew J. Wood, Kenneth H. Fischbeck

    Published 2021
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