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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Príomhchruthaitheoirí: Jakub Malcher, Leonie Heidt, Aurélie Goyenvalle, Helena Escobar, Andreas Marg, Cyriaque Beley, Rachid Benchaouir, Michael Bader, Simone Spuler, Luis García, Verena Schöwel
Formáid: Alt
Teanga:English
Foilsithe / Cruthaithe: Elsevier 2018-12-01
Sraith:Molecular Therapy: Nucleic Acids
Rochtain ar líne:http://www.sciencedirect.com/science/article/pii/S2162253118302257

Ar líne

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

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