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
International Seminar Programme
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