Exon Skipping in a Dysf-Missense Mutant Mouse Model

Exon Skipping in a Dysf-Missense Mutant Mouse Model

Limb girdle muscular dystrophy 2B (LGMD2B) is without treatment and caused by mutations in the dysferlin gene (DYSF). One-third is missense mutations leading to dysferlin aggregation and amyloid formation, in addition to defects in sarcolemmal repair and progressive muscle wasting. Dysferlin-null mo...

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Bibliographic Details
Main Authors: Jakub Malcher, Leonie Heidt, Aurélie Goyenvalle, Helena Escobar, Andreas Marg, Cyriaque Beley, Rachid Benchaouir, Michael Bader, Simone Spuler, Luis García, Verena Schöwel
Format: Article
Language:English
Published: Elsevier 2018-12-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253118302257

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http://www.sciencedirect.com/science/article/pii/S2162253118302257

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