期刊
NUCLEIC ACID THERAPEUTICS
卷 28, 期 3, 页码 119-127出版社
MARY ANN LIEBERT, INC
DOI: 10.1089/nat.2017.0709
关键词
sinusoidal endothelial cells; Stabilins; hepatocytes; asialoglycoprotein receptor
Oligonucleotide therapeutics have emerged as a third distinct platform for drug discovery within the pharmaceutical industry. Five oligonucleotide-based drugs have been approved by the US FDA and over 100 oligonucleotides drugs are currently at different stages of human trials. Several of these oligonucleotide drugs are modified using the phosphorothioate (PS) backbone modification where one of the nonbridging oxygen atoms of the phosphodiester linkage is replaced with sulfur. In this review, we summarize our knowledge on receptor-mediated uptake of PS antisense oligonucleotides (ASOs) within different cell types of the livera privileged organ for the discovery of oligonucleotide-based therapeutics.
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