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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.
Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF, Riney S, Booten SL, Murray SF, Gaus H, Crosby J, Lima WF, Guo S, Monia BP, Swayze EE, Seth PP. Prakash TP, et al. Among authors: low a. Nucleic Acids Res. 2014 Jul;42(13):8796-807. doi: 10.1093/nar/gku531. Epub 2014 Jul 3. Nucleic Acids Res. 2014. PMID: 24992960 Free PMC article.
Comprehensive Structure-Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes.
Prakash TP, Yu J, Migawa MT, Kinberger GA, Wan WB, Østergaard ME, Carty RL, Vasquez G, Low A, Chappell A, Schmidt K, Aghajan M, Crosby J, Murray HM, Booten SL, Hsiao J, Soriano A, Machemer T, Cauntay P, Burel SA, Murray SF, Gaus H, Graham MJ, Swayze EE, Seth PP. Prakash TP, et al. Among authors: low a. J Med Chem. 2016 Mar 24;59(6):2718-33. doi: 10.1021/acs.jmedchem.5b01948. Epub 2016 Mar 9. J Med Chem. 2016. PMID: 26914862
Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins.
Migawa MT, Shen W, Wan WB, Vasquez G, Oestergaard ME, Low A, De Hoyos CL, Gupta R, Murray S, Tanowitz M, Bell M, Nichols JG, Gaus H, Liang XH, Swayze EE, Crooke ST, Seth PP. Migawa MT, et al. Among authors: low a. Nucleic Acids Res. 2019 Jun 20;47(11):5465-5479. doi: 10.1093/nar/gkz247. Nucleic Acids Res. 2019. PMID: 31034558 Free PMC article.
Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates.
Østergaard ME, Jackson M, Low A, E Chappell A, G Lee R, Peralta RQ, Yu J, Kinberger GA, Dan A, Carty R, Tanowitz M, Anderson P, Kim TW, Fradkin L, Mullick AE, Murray S, Rigo F, Prakash TP, Bennett CF, Swayze EE, Gaus HJ, Seth PP. Østergaard ME, et al. Among authors: low a. Nucleic Acids Res. 2019 Jul 9;47(12):6045-6058. doi: 10.1093/nar/gkz360. Nucleic Acids Res. 2019. PMID: 31076766 Free PMC article.
Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle.
Prakash TP, Mullick AE, Lee RG, Yu J, Yeh ST, Low A, Chappell AE, Østergaard ME, Murray S, Gaus HJ, Swayze EE, Seth PP. Prakash TP, et al. Among authors: low a. Nucleic Acids Res. 2019 Jul 9;47(12):6029-6044. doi: 10.1093/nar/gkz354. Nucleic Acids Res. 2019. PMID: 31127296 Free PMC article.
869 results