Refined Design and Liquid-Phase Assembly of GalNAc-siRNA Conjugates: Comparative Efficiency Validation in PCSK9 Targeting.

ABSTRACT: The development and application of therapeutic oligonucleotides, such as siRNA, miRNA, ASOs and aptamers, is a rapidly growing field in biomedicine. These molecules are undergoing extensive preclinical and clinical testing, and the market for synthetic RNA drugs is expanding. However, several challenges remain, including targeted delivery and high costs associated with development, screening and production. One significant advance has been the creation of GalNAc-conjugates, which selectively target ASGPR and deliver oligonucleotides to hepatocytes. Although these conjugates have shown promising results, their widespread use is limited by the lack of effective synthesis methods. Thus, the development of new methods for the synthesis of ligand-oligonucleotide conjugates is an important task

SUBMITTER: Dmitriev NA 

PROVIDER: S-EPMC12899625 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature