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Article

Winkelsas, AM, Grunseich, C, Harmison, GG, Chwalenia, K, Rinaldi, C, Hammond, SM, Johnson, K, Bowerman, M ORCID: https://orcid.org/0000-0002-3579-6403, Arya, S, Talbot, K, Wood, MJ and Fischbeck, KH (2021) Targeting the 5’ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy. Molecular Therapy: Nucleic Acids, 23. pp. 731-742.

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