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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Detalles Bibliográficos
Autores principales: Malcher, Jakub, Heidt, Leonie, Goyenvalle, Aurélie, Escobar, Helena, Marg, Andreas, Beley, Cyriaque, Benchaouir, Rachid, Bader, Michael, Spuler, Simone, García, Luis, Schöwel, Verena
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6172476/
https://www.ncbi.nlm.nih.gov/pubmed/30292141
http://dx.doi.org/10.1016/j.omtn.2018.08.013

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6172476/
https://www.ncbi.nlm.nih.gov/pubmed/30292141
http://dx.doi.org/10.1016/j.omtn.2018.08.013

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