{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T06:37:43Z","timestamp":1790836663989,"version":"4.1.0"},"reference-count":155,"publisher":"Oxford University Press (OUP)","issue":"3","license":[{"start":{"date-parts":[[2025,12,10]],"date-time":"2025-12-10T00:00:00Z","timestamp":1765324800000},"content-version":"vor","delay-in-days":1426,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2022,1,14]],"date-time":"2022-01-14T00:00:00Z","timestamp":1642118400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2022,3,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Genetic cardiomyopathies are disorders of the cardiac muscle, most often explained by pathogenic mutations in genes encoding sarcomere, cytoskeleton, or ion channel proteins. Clinical phenotypes such as heart failure and arrhythmia are classically treated with generic drugs, but aetiology-specific and targeted treatments are lacking. As a result, cardiomyopathies still present a major burden to society, and affect many young and older patients. The Translational Committee of the Heart Failure Association (HFA) and the Working Group of Myocardial Function of the European Society of Cardiology (ESC) organized a workshop to discuss recent advances in molecular and physiological studies of various forms of cardiomyopathies. The study of cardiomyopathies has intensified after several new study setups became available, such as induced pluripotent stem cells, three-dimensional printing of cells, use of scaffolds and engineered heart tissue, with convincing human validation studies. Furthermore, our knowledge on the consequences of mutated proteins has deepened, with relevance for cellular homeostasis, protein quality control and toxicity, often specific to particular cardiomyopathies, with precise effects explaining the aberrations. This has opened up new avenues to treat cardiomyopathies, using contemporary techniques from the molecular toolbox, such as gene editing and repair using CRISPR-Cas9 techniques, antisense therapies, novel designer drugs, and RNA therapies. In this article, we discuss the connection between biology and diverse clinical presentation, as well as promising new medications and therapeutic avenues, which may be instrumental to come to precision medicine of genetic cardiomyopathies.<\/jats:p>","DOI":"10.1002\/ejhf.2414","type":"journal-article","created":{"date-parts":[[2021,12,30]],"date-time":"2021-12-30T17:29:08Z","timestamp":1640885348000},"page":"406-420","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":53,"title":["Targeted Therapies in Genetic Dilated and Hypertrophic Cardiomyopathies: From Molecular Mechanisms to Therapeutic Targets. A Position Paper from the Heart Failure Association (HFA) and the Working Group on Myocardial Function of the European Society of Cardiology (ESC)"],"prefix":"10.1093","volume":"24","author":[{"given":"Rudolf A.","family":"de Boer","sequence":"first","affiliation":[{"name":"Department of Cardiology, University Medical Center Groningen, University of Groningen , Groningen ,","place":["The Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephane","family":"Heymans","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Maastricht University Medical Center (MUMC+) , Maastricht ,","place":["The Netherlands"]},{"name":"Department of Cardiovascular Sciences, University of Leuven , Leuven ,","place":["Belgium"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes","family":"Backs","sequence":"additional","affiliation":[{"name":"Institute of Experimental Cardiology, Heidelberg University , Heidelberg ,","place":["Germany"]},{"name":"German Center for Cardiovascular Research (DZHK), Partner Site Heidelberg\/Mannheim , Heidelberg ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucie","family":"Carrier","sequence":"additional","affiliation":[{"name":"Department of Experimental Pharmacology and Toxicology, University Medical Center Hamburg-Eppendorf , Hamburg ,","place":["Germany"]},{"name":"German Centre for Cardiovascular Research (DZHK), partner site Hamburg\/Kiel\/L\u00fcbeck , Hamburg ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew J.S.","family":"Coats","sequence":"additional","affiliation":[{"name":"Faculty of Medicine, University of Warwick , Warwick ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefanie","family":"Dimmeler","sequence":"additional","affiliation":[{"name":"Institute for Cardiovascular Regeneration, Goethe University , Frankfurt ,","place":["Germany"]},{"name":"German Center for Cardiovascular Research (DZHK) , Frankfurt ,","place":["Germany"]},{"name":"Cardio-Pulmonary Institute (CPI) , Frankfurt ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Eschenhagen","sequence":"additional","affiliation":[{"name":"Department of Experimental Pharmacology and Toxicology, University Medical Center Hamburg-Eppendorf , Hamburg ,","place":["Germany"]},{"name":"German Centre for Cardiovascular Research (DZHK), partner site Hamburg\/Kiel\/L\u00fcbeck , Hamburg ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerasimos","family":"Filippatos","sequence":"additional","affiliation":[{"name":"Department of Cardiology, National and Kapodistrian University of Athens, School of Medicine, Attikon University Hospital , Athens ,","place":["Greece"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lior","family":"Gepstein","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Rambam Health Care Campus, Technion - Israel Institute of Technology , Haifa ,","place":["Israel"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Sebastien","family":"Hulot","sequence":"additional","affiliation":[{"name":"Universit\u00e9 de Paris, INSERM, PARCC , Paris ,","place":["France"]},{"name":"CIC1418 and DMU CARTE, AP-HP, H\u00f4pital Europ\u00e9en Georges-Pompidou , Paris ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ralph","family":"Kn\u00f6ll","sequence":"additional","affiliation":[{"name":"Department of Medicine, Integrated Cardio Metabolic Centre (ICMC), Heart and Vascular Theme, Karolinska Institute , Stockholm ,","place":["Sweden"]},{"name":"Bioscience, Cardiovascular, Renal & Metabolism, BioPharmaceuticals R&D, AstraZeneca , Gothenburg ,","place":["Sweden"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Kupatt","sequence":"additional","affiliation":[{"name":"Department of Cardiology, University Clinic rechts der Isar, Technical University of Munich, Germany and German Center for Cardiovascular Research (DZHK), Munich Heart Alliance , Munich ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang A.","family":"Linke","sequence":"additional","affiliation":[{"name":"Institute of Physiology II, University Hospital Muenster , Muenster ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine E.","family":"Seidman","sequence":"additional","affiliation":[{"name":"Department of Genetics, Harvard Medical School , Boston, MA ,","place":["USA"]},{"name":"Cardiovascular Division, Department of Medicine, Brigham and Women's Hospital , Boston, MA ,","place":["USA"]},{"name":"Howard Hughes Medical Institute, Harvard University , Boston, MA ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. Gabriele","family":"Tocchetti","sequence":"additional","affiliation":[{"name":"Department of Translational Medical Sciences, Center for Basic and Clinical Immunology Research (CISI); Interdepartmental Center for Clinical and Translational Research (CIRCET); Interdepartmental Hypertension Research Center (CIRIAPA), Federico II University , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jolanda","family":"van der Velden","sequence":"additional","affiliation":[{"name":"Department of Physiology, Amsterdam UMC, Amsterdam Cardiovascular Sciences, Vrije Universiteit Amsterdam , Amsterdam ,","place":["The Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roddy","family":"Walsh","sequence":"additional","affiliation":[{"name":"Department of Clinical and Experimental Cardiology, Amsterdam UMC, Amsterdam Cardiovascular Sciences, University of Amsterdam, Heart Center , Amsterdam ,","place":["The 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