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Binding to A Novel Rna-Protein Complex Creates Specificity for Small Molecule Splicing Modifiers to Treat Sma

M. Sivaramakrishnan,K. D. McCarthy, S. Huber,S. Campagne, S. Meier,A. Augustin, T. Heckel, H. Meistermann, M. Hug, P. Birrer,A. Moursy, S. Khawaja, R. Schmucki,N. Berntenis, N. Giroud,S. Golling,M. Tzouros, B. Banfai,G. Duran-Pacheco, J. Lamerz, Y. H. Liu, T. Luebbers,H. Ratni,A. Clery,M. Ebeling,S. Paushkin,A. R. Krainer,F. Allain,F. Metzger

NEUROMUSCULAR DISORDERS（2017）

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