Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates
نویسندگان
چکیده
منابع مشابه
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates
Covalent attachment of a synthetic triantennary N-acetylagalactosamine (GalNAc) ligand to chemically modified siRNA has enabled asialoglycoprotein (ASGPR)-mediated targeted delivery of therapeutically active siRNAs to hepatocytes in vivo. This approach has become transformative for the delivery of RNAi therapeutics as well as other classes of investigational oligonucleotide therapeutics to the ...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2018
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2017.12.021