Base editing effectively prevents early-onset severe cardiomyopathy in Mybpc3 mutant mice

Abstract excerpt

Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder featured by left ventricular (LV) hypertrophy and cardiac dysfunction with an estimated global morbidity of 1:200–1:500. 1 HCM has severe clinical manifestations including heart failure, arrhythmia, sudden cardiac death and stroke. Myosin-binding protein C3 ( MYBPC3 ) mutations account for more than 40% pathogenic variants causing HCM. 2...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cardiomyopathy and Myosin Studies
• Share to CRISPR and Genetic Engineering
• Share to Viral Infections and Immunology Research