Long QT syndrome (LQTS) is a life-threatening congenital arrhythmogenic disease. This study models type-2 LQTS, which is caused by a mutation in the potassium-channel-encoding KCNH2 gene, by using induced pluripotent stem cell technology to generate cardiomyocytes from the fibroblasts of an individual diagnosed with the syndrome. The cells were used to screen a variety of drugs for either amelioration or aggravation of the disease phenotype, demonstrating the power of this approach for drug development and for patient-specific safety screening.
- Ilanit Itzhaki
- Leonid Maizels
- Lior Gepstein
