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-MallebreraYan 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

Research output: Contribution to journalAbstract/Meeting Abstract

Original languageEnglish
Pages (from-to)304-304
Number of pages1
JournalMolecular Therapy
Volume27
Issue number4
Publication statusPublished - 22 Apr 2019
Event22nd Annual Meeting of the American-Society-of-Gene-and-Cell-Therapy (ASGCT) - Washington Hilton, Washington, United States
Duration: 29 Apr 20192 May 2019
https://annualmeeting.asgct.org/UserFiles/file/FinalApprovedVersion_x2.pdf (Meeting guide/program)

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