James G.W. Smith
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
Jamie R. Bhagwan
Diogo Mosqueira email@example.com
James G W Smith
Jamie R Bhagwan
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|
|Journal||Trends in Molecular Medicine|
|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|
Mosqueira Et Al Manuscript TIMM REVISED FINAL2