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)
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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). / de Boer, Rudolf A; Heymans, Stephane; Backs, Johannes; Carrier, Lucie; Coats, Andrew J S; Dimmeler, Stefanie; Eschenhagen, Thomas; Filippatos, Gerasimos; Gepstein, Lior; Hulot, Jean-Sebastien; Knöll, Ralph; Kupatt, Christian; Linke, Wolfgang A; Seidman, Christine E; Tocchetti, C Gabriele; van der Velden, Jolanda; Walsh, Roddy; Seferovic, Petar M; Thum, Thomas.
In: EUR J HEART FAIL, Vol. 24, No. 3, 03.2022, p. 406-420.Research output: SCORING: Contribution to journal › SCORING: Review article › Research
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TY - JOUR
T1 - 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)
AU - de Boer, Rudolf A
AU - Heymans, Stephane
AU - Backs, Johannes
AU - Carrier, Lucie
AU - Coats, Andrew J S
AU - Dimmeler, Stefanie
AU - Eschenhagen, Thomas
AU - Filippatos, Gerasimos
AU - Gepstein, Lior
AU - Hulot, Jean-Sebastien
AU - Knöll, Ralph
AU - Kupatt, Christian
AU - Linke, Wolfgang A
AU - Seidman, Christine E
AU - Tocchetti, C Gabriele
AU - van der Velden, Jolanda
AU - Walsh, Roddy
AU - Seferovic, Petar M
AU - Thum, Thomas
N1 - This article is protected by copyright. All rights reserved.
PY - 2022/3
Y1 - 2022/3
N2 - 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.
AB - 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.
U2 - 10.1002/ejhf.2414
DO - 10.1002/ejhf.2414
M3 - SCORING: Review article
C2 - 34969177
VL - 24
SP - 406
EP - 420
JO - EUR J HEART FAIL
JF - EUR J HEART FAIL
SN - 1388-9842
IS - 3
ER -