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Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.
Viney NJ, Guo S, Tai LJ, Baker BF, Aghajan M, Jung SW, Yu RZ, Booten S, Murray H, Machemer T, Burel S, Murray S, Buchele G, Tsimikas S, Schneider E, Geary RS, Benson MD, Monia BP. Viney NJ, et al. Among authors: burel s. ESC Heart Fail. 2021 Feb;8(1):652-661. doi: 10.1002/ehf2.13154. Epub 2020 Dec 7. ESC Heart Fail. 2021. PMID: 33283485 Free PMC article. Clinical Trial.
Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys.
Burel SA, Han SR, Lee HS, Norris DA, Lee BS, Machemer T, Park SY, Zhou T, He G, Kim Y, MacLeod AR, Monia BP, Lio S, Kim TW, Henry SP. Burel SA, et al. Nucleic Acid Ther. 2013 Jun;23(3):213-27. doi: 10.1089/nat.2013.0422. Nucleic Acid Ther. 2013. PMID: 23692080
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. 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.
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. 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
The Distinct and Cooperative Roles of Toll-Like Receptor 9 and Receptor for Advanced Glycation End Products in Modulating In Vivo Inflammatory Responses to Select CpG and Non-CpG Oligonucleotides.
Paz S, Hsiao J, Cauntay P, Soriano A, Bai L, Machemer T, Xiao X, Guo S, Hung G, Younis H, Bennett CF, Henry S, Yun TJ, Burel S. Paz S, et al. Among authors: burel s. Nucleic Acid Ther. 2017 Oct;27(5):272-284. doi: 10.1089/nat.2017.0668. Epub 2017 Jun 12. Nucleic Acid Ther. 2017. PMID: 28605247
Underlying Immune Disorder May Predispose Some Transthyretin Amyloidosis Subjects to Inotersen-Mediated Thrombocytopenia.
Narayanan P, Curtis BR, Shen L, Schneider E, Tami JA, Paz S, Burel SA, Tai LJ, Machemer T, Kwoh TJ, Xia S, Shattil SJ, Witztum JL, Engelhardt JA, Henry SP, Monia BP, Hughes SG. Narayanan P, et al. Among authors: burel sa. Nucleic Acid Ther. 2020 Apr;30(2):94-103. doi: 10.1089/nat.2019.0829. Epub 2020 Feb 11. Nucleic Acid Ther. 2020. PMID: 32043907 Clinical Trial.
Safety, Pharmacokinetic, and Pharmacodynamic Evaluation of a 2'-(2-Methoxyethyl)-D-ribose Antisense Oligonucleotide-Triantenarry N-Acetyl-galactosamine Conjugate that Targets the Human Transmembrane Protease Serine 6.
Zanardi TA, Korbmacher B, Boone L, Engelhardt JA, Wang Y, Burel S, Prill B, Aghajan M, Guo S, Henry SP. Zanardi TA, et al. Among authors: burel s. J Pharmacol Exp Ther. 2021 Apr;377(1):51-63. doi: 10.1124/jpet.120.000222. Epub 2021 Jan 11. J Pharmacol Exp Ther. 2021. PMID: 33431610
46 results