Phenotypic Screening on iPSC-Derived Disease Models

3 Phenotypic Screening on iPSC-Derived Disease Models 

Phenotypic screening on these disease models is among the most exciting applications in drug discovery because these cells are able to recapitulate known pathophysiological mechanisms. As described previously, only some 

Cardiovascular Long QT syndrome Arrhythmr enicity in cardiac cells treatment with ranolazine rescues arrhythmia [92] 

Neurological Spinal muscular atrophy Reduced SMN levels in iPSCs, reduced size and number of motor neurons rescue phenotype in hiPSCs with valproic acid [93] 

Liver Familial hypercholesterolaemia iPSC-derived hepatocytes have an impaired ability to incorporate LDL [100] 

---