Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice

• Main Authors: Rosie Z Yu (Author), Mark J Graham (Author), Noah Post (Author), Stan Riney (Author), Thomas Zanardi (Author), Shannon Hall (Author), Jennifer Burkey (Author), Colby S Shemesh (Author), Thazha P Prakash (Author), Punit P Seth (Author), Eric E Swayze (Author), Richard S Geary (Author), Yanfeng Wang (Author), Scott Henry (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Triantennary N-acetyl galactosamine (GalNAc3)-conjugated antisense oligonucleotides (ASOs) have greatly improved potency via receptor-mediated uptake. In the present study, the in vivo pharmacology of a 2'-O-(2-methoxyethyl)-modified ASO conjugated with GalNAc3 (ISIS 681257) together with its u...