Roger J. Hajjar

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.

This study shows that AAV1.SERCA2a gene therapy improves heart function and reduces arrhythmia in a pig model of Duchenne muscular dystrophy.

This paper demonstrates that allele-specific RNAi therapy reduces disease-causing mutations in a mouse model of restrictive cardiomyopathy, improving cardiac function and reducing pathological markers.