Ontology highlight
ABSTRACT:
SUBMITTER: Janas MM
PROVIDER: S-EPMC5818625 | biostudies-literature | 2018 Feb
REPOSITORIES: biostudies-literature
Janas Maja M MM Schlegel Mark K MK Harbison Carole E CE Yilmaz Vedat O VO Jiang Yongfeng Y Parmar Rubina R Zlatev Ivan I Castoreno Adam A Xu Huilei H Shulga-Morskaya Svetlana S Rajeev Kallanthottathil G KG Manoharan Muthiah M Keirstead Natalie D ND Maier Martin A MA Jadhav Vasant V
Nature communications 20180219 1
Small interfering RNAs (siRNAs) conjugated to a trivalent N-acetylgalactosamine (GalNAc) ligand are being evaluated in investigational clinical studies for a variety of indications. The typical development candidate selection process includes evaluation of the most active compounds for toxicity in rats at pharmacologically exaggerated doses. The subset of GalNAc-siRNAs that show rat hepatotoxicity is not advanced to clinical development. Potential mechanisms of hepatotoxicity can be associated w ...[more]