High affinity antisense oligonucleotides (ASOs) containing bicylic modifications (BNA) such as locked nucleic acid (LNA) or constrained ethyl (cEt) designed to induce target RNA cleavage have been shown to have enhanced potency along with a higher propensity to cause hepatotoxicity. In order to unravel the mechanism of this hepatotoxicity, we leveraged GeneSpring GX analysis software to analyze transcriptional profiles from the livers of mice treated with a panel of highly efficacious hepatotoxic or non-hepatotoxic LNA ASOs.
Speaker:
Sebastien A. Burel, PhD
Director, Nonclinical Development, Ionis Pharmaceuticals, California
Details:
14 June 2017, 8 AM PST
