Potent and durable gene modulation in heart and muscle with chemically defined siRNAs
Hassan H. Fakih, Clemens Lochmann, Rosemary Gagnon, Ashley Summers, Jillian Caiazzi, Julianna E. Buchwald, Qi Tang, Bruktawit Maru, Samuel R. Hildebrand, Mohammad Zain UI Abideen, Raymond C. Furgal, Katherine Y. Gross, Yeon-Suk Yang, David Cooper, Kathryn R. Monopoli, , Anastasia Khvorova
This study develops chemically defined siRNAs for durable gene modulation in muscle and heart, showing long-term silencing and functional benefits with minimal toxicity.
Nucleic Acids Research · Nov 26, 2025Peer-reviewedPharmacology & Toxicology