Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

Tatiana O. Gavrilina, Vicki L. McGovern, Eileen Workman, Thomas O. Crawford, Rocky G. Gogliotti, Christine J. Didonato, Umrao R. Monani, Glenn E. Morris, Arthur H.M. Burghes

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Medicine & Life Sciences