Raffaele COPPINI

Department of Neuroscience, Psychology, Drug sciences and Child Health (NeuroFarBa), University of Florence and Careggi University Hospital, Florence, ITALY

Cellular and molecular targets for drug therapy in hypertrophic cardiomyopathy

Host: S. Zacchigna

Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiac disease, and is associated

with an increased risk of life-threatening arrhythmias and heart failure. No current pharmacological

options are capable of addressing the risk of arrhythmias and reducing the risk of disease progres-

sion. Preclinical studies in HCM are limited by the low translational potential of transgenic mouse

models. Therefore, our group studied the molecular and cellular mechanisms of arrhythmogenicity

and mechanical dysfunction in left ventricular samples from patients with HCM undergoing surgical

septal myectomy. Moreover, we recently employed lines of induced pluripotent stem cells obtained

from HCM patients carrying relevant mutations to produce in vitro cellular models of disease. Both

these approaches were used to identify and validate new molecular targets as well as to test novel

therapeutic options for this disease

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