Reversal of contractile defects by mediating calcium homeostasis in human mini-heart models of heart failure with preserved ejection fraction (HFpEF) leads to first-in-human gene therapy clinical trial
Kevin D. Costa, Andy O.-T. Wong, Suet Yee Mak, Erin G. Roberts, Wendy Keung, Cláudia Correia, Anna Walentinsson, Jonas Christoffersson, Alice Cheung, Deborah K. Lieu, Karin Jennbacken, Qing‐Dong Wang, Roger J. Hajjar, Ronald A. Li
This paper develops human mini-heart models of HFpEF, identifies SERCA2a downregulation, and demonstrates gene therapy efficacy, leading to a first-in-human clinical trial.
bioRxiv · Aug 27, 2024 · PreprintBioengineering