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Phenotypic Drug Screening for Dysferlinopathy Using Patient‐Derived Induced Pluripotent Stem Cells

Dysferlinopathy is a progressive muscle disorder that includes limb‐girdle muscular dystrophy type 2B and Miyoshi myopathy (MM). It is caused by mutations in the dysferlin (DYSF) gene, whose function is to reseal the muscular membrane. Treatment with proteasome inhibitor MG‐132 has been shown to inc...

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Detalles Bibliográficos
Autores principales:	Kokubu, Yuko, Nagino, Tomoko, Sasa, Katsunori, Oikawa, Tatsuo, Miyake, Katsuya, Kume, Akiko, Fukuda, Mikiko, Fuse, Hiromitsu, Tozawa, Ryuichi, Sakurai, Hidetoshi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2019
Materias:	Cell‐Based Drug Development, Screening, and Toxicology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6766604/ https://www.ncbi.nlm.nih.gov/pubmed/31250983 http://dx.doi.org/10.1002/sctm.18-0280

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