Mouse genome-wide association studies and systems genetics uncover the genetic architecture associated with hepatic pharmacokinetic and pharmacodynamic properties of a constrained ethyl antisense oligonucleotide targeting Malat1.

Antisense oligonucleotides (ASOs) have demonstrated variation of efficacy in patient populations. This has prompted our investigation into the contribution of genetic architecture to ASO pharmacokinetics (PK) and pharmacodynamics (PD). Genome wide association (GWA) and transcriptomic analysis in a h...

Full description

Bibliographic Details
Main Authors: Elaine Pirie, Shayoni Ray, Calvin Pan, Wuxia Fu, Andrew F Powers, Danielle Polikoff, Colton M Miller, Katrina M Kudrna, Edward N Harris, Aldons J Lusis, Rosanne M Crooke, Richard G Lee
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2018-10-01
Series:PLoS Genetics
Online Access:http://europepmc.org/articles/PMC6224167?pdf=render