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Personalized medicine in the dish to prevent calcium leak associated with short-coupled polymorphic ventricular tachycardia in patient-derived cardiomyocytes.

Yvonne SleimanSteven ReikenAzzouz CharrabiFabrice JaffréLeah R SittenfeldJean-Luc PasquiéSarah ColombaniBruce B LermanShuibing ChenAndrew R MarksJim W CheungTodd EvansAlain LacampagneAlbano C Meli
Published in: Stem cell research & therapy (2023)
release and contractile properties in RyR2-H29D hiPSC-CMs, whereas the effect of propranolol is partial, and verapamil appears ineffective. In contrast with the 3 other drugs, S107 was able to prevent a major post-translational modification of RyR2-H29D mutant channels, the loss of calstabin2 binding to RyR2. Using patient-specific hiPSC and CRISPR/Cas9 technologies, we showed that S107 is the most efficient in vitro candidate for treating the short-coupled PMVT at rest.
Keyphrases
  • crispr cas
  • genome editing
  • magnetic resonance
  • skeletal muscle
  • magnetic resonance imaging
  • smooth muscle
  • endothelial cells
  • wild type
  • high glucose