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Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.
Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS, Krainer AR, Henry SP, Bennett CF. Rigo F, et al. Among authors: gaus h. J Pharmacol Exp Ther. 2014 Jul;350(1):46-55. doi: 10.1124/jpet.113.212407. Epub 2014 Apr 30. J Pharmacol Exp Ther. 2014. PMID: 24784568 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: gaus hj. Nucleic Acids Res. 2019 Jul 9;47(12):6045-6058. doi: 10.1093/nar/gkz360. Nucleic Acids Res. 2019. PMID: 31076766 Free PMC article.
Peptide nucleic acids conjugated to short basic peptides show improved pharmacokinetics and antisense activity in adipose tissue.
Wancewicz EV, Maier MA, Siwkowski AM, Albertshofer K, Winger TM, Berdeja A, Gaus H, Vickers TA, Bennett CF, Monia BP, Griffey RH, Nulf CJ, Hu J, Corey DR, Swayze EE, Kinberger GA. Wancewicz EV, et al. Among authors: gaus h. J Med Chem. 2010 May 27;53(10):3919-26. doi: 10.1021/jm901489k. J Med Chem. 2010. PMID: 20420385 Free PMC article.
Sequence-specific 2'-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of platelet-leukocyte aggregates.
Slingsby MHL, Vijey P, Tsai IT, Roweth H, Couldwell G, Wilkie AR, Gaus H, Goolsby JM, Okazaki R, Terkovich BE, Semple JW, Thon JN, Henry SP, Narayanan P, Italiano JE Jr. Slingsby MHL, et al. Among authors: gaus h. Haematologica. 2022 Feb 1;107(2):519-531. doi: 10.3324/haematol.2020.260059. Haematologica. 2022. PMID: 33567808 Free PMC article.
69 results