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Duque, Sandra I; Arnold, W David; Odermatt, Philipp; Li, Xiaohui; Porensky, Paul N; Schmelzer, Leah; Meyer, Kathrin; Kolb, Stephen J; Schümperli, Daniel; Kaspar, Brian K; Burghes, Arthur H M (2015). A large animal model of spinal muscular atrophy and correction of phenotype. Annals of neurology, 77(3), pp. 399-414. Wiley-Blackwell 10.1002/ana.24332