Conference Proceedings

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 Show all

MOLECULAR THERAPY | CELL PRESS | Published : 2019