Targeting the 5 0 untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
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K. Fischbeck | M. Bowerman | K. Talbot | S. Hammond | M. Wood | C. Grunseich | Kory R. Johnson | Katarzyna Chwalenia | G. Harmison | Carlo Rinaldi | Audrey M. Winkelsas | S. Arya | C. Rinaldi