Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates
Author:
Affiliation:
1. Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA
Funder
Ionis Pharmaceuticals
Publisher
Oxford University Press (OUP)
Subject
Genetics
Link
http://academic.oup.com/nar/advance-article-pdf/doi/10.1093/nar/gkz360/28599303/gkz360.pdf
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3. Cellular uptake and trafficking of antisense oligonucleotides;Crooke;Nat. Biotech.,2017
4. Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice;Prakash;Nucleic Acids Res.,2014
5. Integrated assessment of the clinical performance of GalNAc3-Conjugated 2′-O-Methoxyethyl chimeric antisense oligonucleotides: I.Human Volunteer Experience;Crooke;Nucleic Acid Ther.,2019
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