A Common Deep Intronic Mutation Causing Collagen VI-Related Muscular Dystrophy: Validation of Splice-Modulating Approaches in Vitro and Development of a Mouse Model Veronique Bolduc , A. Reghan Foley , Herimela Solomon-Degefa , Apurva Sarathy , Sandra Donkervoort , Ying Hu , Grace S. Chen , Matthew Nalls , Haiyan Zhou , Sara Aguti , Beryl B. Cummings , Monkol Lek , Taru Tukiainen , Jamie L. Marshall , Oded Regev , Dina Marek-Yagel , Anna Sarkozy , Russell J. Butterfield , Cristina Jou , Cecilia Jimenez-Mallebrera , Yan Li , Jun Cheng , Lisa Garrett , Corine Gartioux , Kamel Mamchaoui , Valerie Allamand , Francesca Gualandi , Alessandra Ferlini , Eric Hanssen , Steve D. Wilton , Shireen R. Lamande , Daniel G. MacArthur , Raimund Wagener , Francesco Muntoni , Carsten G. Bonnemann MOLECULAR THERAPY（2019）
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