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Generation of two genomic-integration-free DMD iPSC lines with mutations affecting all dystrophin isoforms and potentially amenable to exon-skipping

Duchenne muscular dystrophy (DMD) is the most common paediatric muscular dystrophy and is caused by mutations in the DYSTROPHIN gene. We generated two induced pluripotent stem cell (iPSC) lines from DMD patients with nonsense mutations in exons 68 (UCLi011-A) or 70 (UCLi012-A) by transfecting reprog...

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Detalles Bibliográficos
Autores principales:	Ferrari, Giulia, Muntoni, Francesco, Tedesco, Francesco Saverio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7057262/ https://www.ncbi.nlm.nih.gov/pubmed/32087527 http://dx.doi.org/10.1016/j.scr.2019.101688

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