Therapeutic targeting of the Platelet-Derived Growth Factor (PDGF) signaling pathway in dilated cardiomyopathy

Dilated cardiomyopathy (DCM) is principally characterized by left ventricular enlargement and/or a reduction in systolic function. In many cases for individuals with DCM, no etiology can be determined, therefore clinicians should suspect that a pathogenic variant of the LMNA gene may be the underlying cause. Initial signs of LMNA-related DCM are sudden cardiac arrest leading to death, owing to prevalence of arrythmias in the individual. Researchers at Stanford University have developed a novel approach to the treatment and prevention of proarrhythmic in LMNA-related DCM by establishing the link between hyperactivation of signaling pathways and the LMNA gene in patient-specific induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), and subsequent treatment using tyrosine kinase inhibitors.