Promise and potential peril with lumacaftor for the trafficking defective type 2 long- qt syndrome-causative variants, p.G604S, p.N633S, and p.R685P, using patient-specific re-engineered cardiomyocytes

Medicine & Life Sciences

• lumacaftor 100%
• Cardiac Myocytes 51%
• Induced Pluripotent Stem Cells 16%
• Mutation 13%
• Long Qt Syndrome 2 8%
• Long QT Syndrome 6%
• Potassium Channels 5%
• Ether 5%
• Protein Transport 5%
• United States Food and Drug Administration 4%
• Cystic Fibrosis 4%
• Action Potentials 4%
• Coloring Agents 4%
• Therapeutics 4%
• Phenotype 2%
• Genes 2%