ROZEMA, DAVID B.,WAKEFIELD, DARREN H.,BLOKHIN, ANDREI V.,BENSON, JONATHAN D.,LI, ZHEN,PEI, TAO,FLEITZ, FRED
申请号：
CA3011946
公开号：
CA3011946A1
申请日:
2017.03.07
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds, that are useful in directing the compounds to the target in vivo. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to an expression-inhibiting oligomeric compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.
