Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of MYBPC3-Associated Cardiomyopathy
Amara Greer-Short, Anna Greenwood, Elena C. Leon, Tawny Neal Qureshi, Konor von Kraut, Justin Wong, Jonathan H. Tsui, Christopher A. Reid, Ze Cheng, Emilee Easter, Jin Yang, Jaclyn J. Ho, Stephanie S. Steltzer, Ana Budan, Marie Cho, , Laura Marie Lombardi
This study demonstrates that AAV gene therapy with TN-201 restores MYBPC3 protein levels, reverses cardiac hypertrophy, and improves survival in a murine model of MYBPC3-associated HCM.
Nature Communications · Mar 4, 2025Peer-reviewedGenetics