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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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Detaylı Bibliyografya
Yayımlandı:Stem Cell Res
Asıl Yazarlar: Ferrari, Giulia, Muntoni, Francesco, Tedesco, Francesco Saverio
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Elsevier 2020
Konular:
Online Erişim: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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