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Modeling Hypertrophic Cardiomyopathy: Mechanistic Insights and Pharmacological Intervention

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


James G.W. Smith

Jamie R. Bhagwan

Diogo Mosqueira

James G W Smith

Jamie R Bhagwan

Chris Denning


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.

Journal Article Type Article
Publication Date 2019-07
Journal Trends in Molecular Medicine
Print ISSN 1471-4914
Electronic ISSN 1471-499X
Publisher Elsevier
Peer Reviewed Peer Reviewed
Institution 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, doi:10.1016/j.molmed.2019.06.005
Keywords Hypertrophic cardiomyopathy; Disease modeling; HCM mechanisms
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