Human iPSCs use as preclinical model for drug study in cerebrovascular diseases

Abstract

Stroke is the main cause of mortality and morbidity in developed countries. A relevant percentage of strokes is due to several cerebrovascular pathologies with genetic background. While the study of these diseases in animal models is proving inefficient, with the development of the cellular reprogramming technology in 2006, a new approach appeared to overcome the existing barriers found in these models. Thanks to the cellular reprogramming technology, the current thesis supposes the first time that an in vitro cellular model with the patient's own cells is achieved for the study of one of these cerebrovascular pathologies, CADASIL, focusing on the abnormal behavior of the mutated protein NOTCH3.