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Antisense oligonucleotides containing conformationally constrained 2',4'-(N-methoxy)aminomethylene and 2',4'-aminooxymethylene and 2'-O,4'-C-aminomethylene bridged nucleoside analogues show improved potency in animal models.
Prakash TP, Siwkowski A, Allerson CR, Migawa MT, Lee S, Gaus HJ, Black C, Seth PP, Swayze EE, Bhat B. Prakash TP, et al. Among authors: lee s. J Med Chem. 2010 Feb 25;53(4):1636-50. doi: 10.1021/jm9013295. J Med Chem. 2010. PMID: 20108935
Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 3'-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides.
Egli M, Pallan PS, Allerson CR, Prakash TP, Berdeja A, Yu J, Lee S, Watt A, Gaus H, Bhat B, Swayze EE, Seth PP. Egli M, et al. Among authors: lee s. J Am Chem Soc. 2011 Oct 19;133(41):16642-9. doi: 10.1021/ja207086x. Epub 2011 Sep 22. J Am Chem Soc. 2011. PMID: 21919455 Free PMC article.
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: lee s. 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.
Amphoteric liposomes enable systemic antigen-presenting cell-directed delivery of CD40 antisense and are therapeutically effective in experimental arthritis.
Andreakos E, Rauchhaus U, Stavropoulos A, Endert G, Wendisch V, Benahmed AS, Giaglis S, Karras J, Lee S, Gaus H, Bennett CF, Williams RO, Sideras P, Panzner S. Andreakos E, et al. Among authors: lee s. Arthritis Rheum. 2009 Apr;60(4):994-1005. doi: 10.1002/art.24434. Arthritis Rheum. 2009. PMID: 19333921 Free article.
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