Amelioration of intracellular Ca regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes.

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ID: 57800
2019
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Ranked #5 of 66 articles by views in Biochemical and biophysical research communications

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Abstract
Duchenne muscular dystrophy (DMD) is a devastating muscle disorder caused by frameshift mutations in the DMD gene. DMD involves cardiac muscle, and the presence of ventricular arrhythmias or congestive failure is critical for prognosis. Several novel therapeutic approaches are being evaluated in ongoing clinical trials. Among them, exon-skipping therapy to correct frameshift mutations with antisense oligonucleotides is promising; however, their therapeutic efficacies on cardiac muscle in vivo remain unknown. In this study, we established induced-pluripotent stem cells (iPSCs) from T cells from a DMD patient carrying a DMD-exon 46-55 deletion, differentiated the iPSCs into cardiomyocytes, and treated them with phosphorodiamidate morpholino oligomers. The efficiency of exon-45 skipping increased in a dose-dependent manner and enabled restoration of the DMD gene product, dystrophin. Further, Ca-imaging analysis showed a decreased number of arrhythmic cells and improved transient Ca signaling after exon skipping. Thus, exon-45 skipping may be effective for cardiac involvement in DMD patients harboring the DMD-exon 46-55 deletion.
Reference Key
sato2019ameliorationbiochemical Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Sato, Mitsuto;Shiba, Naoko;Miyazaki, Daigo;Shiba, Yuji;Echigoya, Yusuke;Yokota, Toshifumi;Takizawa, Hotake;Aoki, Yoshitsugu;Takeda, Shin'ichi;Nakamura, Akinori;
Journal Biochemical and biophysical research communications
Year 2019
DOI
S0006-291X(19)31825-X
URL
Keywords
cardiomyocyte arrhythmic cell ca(2+) transient duchenne muscular dystrophy exon skipping induced-pluripotent stem cell

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