Allele-specific silencing ameliorates restrictive cardiomyopathy due to a human myosin regulatory light chain mutation
Kathia Zaleta-Rivera, Alexandra Dainis, Alexandre J. S. Ribeiro, Pablo Cordero, Gabriel R. Rubio, Ching Shang, Jing Liu, Thomas Finsterbach, Victoria Nicole Parikh, Shirley C. Sutton, Kinya Seo, Nikita Sinha, Nikhil Jain, Yong Huang, Roger J. Hajjar, , Euan Angus Ashley
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.
Circulation · Jul 18, 2019Peer-reviewedMolecular Biology