Key result
Sarcomeric defects drive contractile dysfunction in inherited cardiomyopathies, necessitating novel sarcomere modulators.
Why the study?
Inherited cardiomyopathies cause contractile dysfunction with no disease-specific treatments currently available, requiring a better understanding of sarcomeric defects and cardiomyocyte mechanics to develop novel therapies.
Understanding the molecular basis of inherited cardiomyopathies and mutation-induced alterations in cardiomyocyte mechanics opens new avenues for targeted therapies modulating sarcomere contractility.
Supports targeted sarcomere modulation in inherited cardiomyopathies; leaves open clinical translation of novel agents.
Inherited cardiomyopathies form a heterogenous group of disorders that affect the structure and function of the heart. Defects in the genes encoding sarcomeric proteins are associated with various perturbations that induce contractile dysfunction and promote disease development. In this review we aimed to outline the functional consequences of the major inherited cardiomyopathies in terms of myocardial contraction and kinetics, and to highlight the structural and functional alterations in some sarcomeric variants that have been demonstrated to be involved in the pathogenesis of the inherited cardiomyopathies. A particular focus was made on mutation-induced alterations in cardiomyocyte mechanics. Since no disease-specific treatments for familial cardiomyopathies exist, several novel agents have been developed to modulate sarcomere contractility. Understanding the molecular basis of the disease opens new avenues for the development of new therapies. Furthermore, the earlier the awareness of the genetic defect, the better the clinical prognostication would be for patients and the better the prevention of development of the disease.
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Hassoun et al. (2021) conducted a review in Inherited cardiomyopathies. Inherited cardiomyopathies involve sarcomeric protein defects that induce contractile dysfunction, highlighting the need for novel agents to modulate sarcomere contractility.
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