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

    Udgivet 2021
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