@article{b1d694aa8a6a491795d5e3d4ec038867,
title = "Targets for therapy in sarcomeric cardiomyopathies",
abstract = "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.",
keywords = "Dilated cardiomyopathy, Energetics and microvasculature, Gene therapy, Hypertrophic cardiomyopathy, Ion channels",
author = "Tardiff, {Jil C.} and Lucie Carrier and Bers, {Donald M.} and Corrado Poggesi and Cecilia Ferrantini and Raffaele Coppini and Maier, {Lars S.} and Houman Ashrafian and Sabine Huke and {Van Der Velden}, Jolanda",
note = "Funding Information: L.S.M. acknowledges research grants and funding from CVT, GILEAD, and MENARINI/Berlin-Chemie. L.S.M. is funded by Deutsche Forschungsge-meinschaft (DFG) GRK 1816 RP3, by the DZHK (Deutsches Zentrum f{\"u}r Herz-Kreislauf-Forschung), and the Leducq Foundation. L.C. is supported by grants from the DZHK (German Centre for Cardiovascular Research) and the German Ministry of Research and Education (BMBF), the Leducq Foundation (research grant no. 11, CVD 04), and Association Institut de Myologie (Paris). C.P. is supported by Telethon Italy grant GGP13162. H.A. is supported by British Heart Foundation, including the BHF centre of research excellence and the MRC. J.v.d.V. is supported by the Netherlands organization for Scientific Research (NWO; VIDI grant 91711344) and by a CVON grant (Cardiovasculair Onderzoek Nederland; ARENA consortium). We further acknowledge support from the 7th Framework Program of the European Union ({\textquoteleft}BIG-HEART{\textquoteright}, grant agreement 241577). J.C.T. is supported by the National Institutes of Health (grants HL075619 and HL107046). Publisher Copyright: {\textcopyright} 2015 Published on behalf of the European Society of Cardiology. All rights reserved. {\textcopyright} The Author 2015.",
year = "2015",
month = apr,
day = "1",
doi = "10.1093/cvr/cvv023",
language = "English (US)",
volume = "105",
pages = "457--470",
journal = "Cardiovascular Research",
issn = "0008-6363",
publisher = "Oxford University Press",
number = "4",
}