Figure - PMC

Skip to main content

An official website of the United States government

Here's how you know

Here's how you know

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

View full-text article in PMC

. 2024 Mar 30;35(2):102184. doi: 10.1016/j.omtn.2024.102184

Search in PMC
Search in PubMed
View in NLM Catalog
Add to search

© 2024 The Authors

This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

PMC Copyright notice

Hepatic delivery systems and action modes of oligonucleotides for liver diseases

GalNAc-ASO/siRNA conjugates are delivered to hepatocytes via the ASGPR expressed on hepatocyte surface. LNPs containing siRNA/miRNA are internalized via the LDLR expressed on the hepatocyte surface. AAV delivers shRNA and miRNA for gene knockdown via primary cell-surface glycoprotein receptors and secondary receptors or universal AAV receptor (AAVR). ASO binds to the target mRNA and attracts RNase H for mRNA degradation. siRNA is loaded into the RISC, leading to targeted mRNA cleavage or translation inhibition. miRNA complementary base pairs with the corresponding miRNA to block the cleavage of targeted mRNA via RISC. GalNAc, N-acetylgalactosamine; LNP, lipid nanoparticle; AAV. adeno-associated virus; LDLR, low-density lipoprotein receptor.