Targets for therapy in sarcomeric cardiomyopathies
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Targets for therapy in sarcomeric cardiomyopathies. / Tardiff, Jil C; Carrier, Lucie; Bers, Donald M; Poggesi, Corrado; Ferrantini, Cecilia; Coppini, Raffaele; Maier, Lars S; Ashrafian, Houman; Huke, Sabine; van der Velden, Jolanda.
In: CARDIOVASC RES, Vol. 105, No. 4, 01.04.2015, p. 457-70.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Targets for therapy in sarcomeric cardiomyopathies
AU - Tardiff, Jil C
AU - Carrier, Lucie
AU - Bers, Donald M
AU - Poggesi, Corrado
AU - Ferrantini, Cecilia
AU - Coppini, Raffaele
AU - Maier, Lars S
AU - Ashrafian, Houman
AU - Huke, Sabine
AU - van der Velden, Jolanda
N1 - Published on behalf of the European Society of Cardiology. All rights reserved. © The Author 2015. For permissions please email: journals.permissions@oup.com.
PY - 2015/4/1
Y1 - 2015/4/1
N2 - To date, no compounds or interventions exist that treat or prevent sarcomeric cardiomyopathies. Established therapies currently improve the outcome, but novel therapies may be able to more fundamentally affect the disease process and course. Investigations of the pathomechanisms are generating molecular insights that can be useful for the design of novel specific drugs suitable for clinical use. As perturbations in the heart are stage-specific, proper timing of drug treatment is essential to prevent initiation and progression of cardiac disease in mutation carrier individuals. In this review, we emphasize potential novel therapies which may prevent, delay, or even reverse hypertrophic cardiomyopathy caused by sarcomeric gene mutations. These include corrections of genetic defects, altered sarcomere function, perturbations in intracellular ion homeostasis, and impaired myocardial energetics.
AB - To date, no compounds or interventions exist that treat or prevent sarcomeric cardiomyopathies. Established therapies currently improve the outcome, but novel therapies may be able to more fundamentally affect the disease process and course. Investigations of the pathomechanisms are generating molecular insights that can be useful for the design of novel specific drugs suitable for clinical use. As perturbations in the heart are stage-specific, proper timing of drug treatment is essential to prevent initiation and progression of cardiac disease in mutation carrier individuals. In this review, we emphasize potential novel therapies which may prevent, delay, or even reverse hypertrophic cardiomyopathy caused by sarcomeric gene mutations. These include corrections of genetic defects, altered sarcomere function, perturbations in intracellular ion homeostasis, and impaired myocardial energetics.
U2 - 10.1093/cvr/cvv023
DO - 10.1093/cvr/cvv023
M3 - SCORING: Journal article
C2 - 25634554
VL - 105
SP - 457
EP - 470
JO - CARDIOVASC RES
JF - CARDIOVASC RES
SN - 0008-6363
IS - 4
ER -