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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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Detalhes bibliográficos
Publicado no:Stem Cell Res
Main Authors: Ferrari, Giulia, Muntoni, Francesco, Tedesco, Francesco Saverio
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7057262/
https://ncbi.nlm.nih.gov/pubmed/32087527
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.scr.2019.101688
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