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Modeling Hypertrophic Cardiomyopathy: Mechanistic Insights and Pharmacological Intervention

Bhagwan, Jamie R.; Smith, James G.W.; Mosqueira, Diogo; Smith, James G W; Bhagwan, Jamie R; Denning, Chris

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Authors

Jamie R. Bhagwan

James G.W. Smith

Diogo Mosqueira

James G W Smith

Jamie R Bhagwan

CHRIS DENNING chris.denning@nottingham.ac.uk
Professor of Stem Cell Biology



Abstract

HCM is a prevalent and complex disease governed by multiple molecular mechanisms, and there is currently no efficient cure. New treatment strategies are under development, and several drugs are reaching the final stages of clinical trials, with partial efficacy.

HCM disease models vary in complexity. Furthermore, they are limited by sample availability, demanding logistics, preparation artefacts, oversimplicity, and species-differences relative to the human cardiovascular physiology, often producing conflicting results.

Application of genome-editing technology to hPSCs enables unlimited isogenic sets of heart cells in which the primary mutation is the only change, allowing elucidation of disease phenotypes and genetic causation.

Uncovering new disease mechanisms and targets will pave the way to more efficient HCM therapeutics.

Citation

Bhagwan, J. R., Smith, J. G., Mosqueira, D., Smith, J. G. W., Bhagwan, J. R., & Denning, C. (2019). Modeling Hypertrophic Cardiomyopathy: Mechanistic Insights and Pharmacological Intervention. Trends in Molecular Medicine, 25(9), 775-790. https://doi.org/10.1016/j.molmed.2019.06.005

Journal Article Type Article
Acceptance Date Jun 19, 2019
Online Publication Date Jul 17, 2019
Publication Date 2019-09
Deposit Date Jul 5, 2019
Publicly Available Date Mar 29, 2024
Journal Trends in Molecular Medicine
Print ISSN 1471-4914
Electronic ISSN 1471-499X
Publisher Elsevier
Peer Reviewed Peer Reviewed
Volume 25
Issue 9
Pages 775-790
DOI https://doi.org/10.1016/j.molmed.2019.06.005
Keywords Hypertrophic cardiomyopathy; Disease modeling; HCM mechanisms
Public URL https://nottingham-repository.worktribe.com/output/2272833
Publisher URL https://www.sciencedirect.com/science/article/pii/S1471491419301674?via%3Dihub

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