A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice

Eileen Workman, Luciano Saieva, Tessa L. Carrel, Thomas O. Crawford, Don Liu, Cathleen Lutz, Christine E. Beattie, Livio Pellizzoni, Arthur H.M. Burghes

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79 Scopus citations

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Medicine & Life Sciences