A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy

Umrao R. Monani, Matthew T. Pastore, Tatiana O. Gavrilina, Sibylle Jablonka, Thanh T. Le, Catia Andreassi, Jennifer M. DiCocco, Christian Lorson, Elliot J. Androphy, Michael Sendtner, Michael Podell, Arthur H.M. Burghes

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