Uncovering Mechanisms of Hepatotoxicity for High Affinity Antisense Oligonucleotides – 3’ end RNA-seq Profiling Using GeneSpring GX
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 Name
Sebastien A. Burel, PhD
Director, Nonclinical Development
Ionis Pharmaceuticals
Carlsbad, California 92010
Session details
Wednesday, June 14, 2017, 8:00 a.m. PST
Register here
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 Name
Sebastien A. Burel, PhD
Director, Nonclinical Development
Ionis Pharmaceuticals
Carlsbad, California 92010
Session details
Wednesday, June 14, 2017, 8:00 a.m. PST
Register here