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Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.
Burel SA, Hart CE, Cauntay P, Hsiao J, Machemer T, Katz M, Watt A, Bui HH, Younis H, Sabripour M, Freier SM, Hung G, Dan A, Prakash TP, Seth PP, Swayze EE, Bennett CF, Crooke ST, Henry SP. Burel SA, et al. Among authors: watt a. Nucleic Acids Res. 2016 Mar 18;44(5):2093-109. doi: 10.1093/nar/gkv1210. Epub 2015 Nov 8. Nucleic Acids Res. 2016. PMID: 26553810 Free PMC article.
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: watt a. 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.
Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.
Østergaard ME, Southwell AL, Kordasiewicz H, Watt AT, Skotte NH, Doty CN, Vaid K, Villanueva EB, Swayze EE, Bennett CF, Hayden MR, Seth PP. Østergaard ME, et al. Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19. Nucleic Acids Res. 2013. PMID: 23963702 Free PMC article.
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.
Lagier-Tourenne C, Baughn M, Rigo F, Sun S, Liu P, Li HR, Jiang J, Watt AT, Chun S, Katz M, Qiu J, Sun Y, Ling SC, Zhu Q, Polymenidou M, Drenner K, Artates JW, McAlonis-Downes M, Markmiller S, Hutt KR, Pizzo DP, Cady J, Harms MB, Baloh RH, Vandenberg SR, Yeo GW, Fu XD, Bennett CF, Cleveland DW, Ravits J. Lagier-Tourenne C, et al. Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9. doi: 10.1073/pnas.1318835110. Epub 2013 Oct 29. Proc Natl Acad Sci U S A. 2013. PMID: 24170860 Free PMC article.
646 results