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Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

Dysferlinopathy is a progressive myopathy caused by mutations in the dysferlin (DYSF) gene. Dysferlin protein plays a major role in plasma-membrane resealing. Some patients with DYSF deletion mutations exhibit mild symptoms, suggesting some regions of DYSF can be removed without significantly impact...

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Detalles Bibliográficos
Autores principales:	Lee, Joshua J.A., Maruyama, Rika, Duddy, William, Sakurai, Hidetoshi, Yokota, Toshifumi
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/PMC6234522/ https://www.ncbi.nlm.nih.gov/pubmed/30439648 http://dx.doi.org/10.1016/j.omtn.2018.10.004

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