Abstract

Disease-specific stem cell therapies, created from induced pluripotent stem cell lines containing the genetic defects responsible for a particular disease, have the potential to revolutionize the treatment of refractory chronic diseases. Given their capacity to differentiate into any human cell type, these cell lines might be reprogrammed to correct a disease-causing genetic defect in any tissue or organ, in addition to offering a more clinically realistic model for testing new drugs and studying disease mechanisms. Clinical translation of these therapies provides an opportunity to design a more systematic, accessible and patient-influenced model for the delivery of medically innovative treatments to chronically ill patients