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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: monia bp. 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.
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: monia bp. 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.
Antisense oligonucleotide therapy for TTR amyloidosis.
Benson MD, Pandey S, Witchell D, Jazayeri A, Siwkowski A, Monia B, Kluve-Beckerman B. Benson MD, et al. Amyloid. 2011 Jun;18 Suppl 1:60. doi: 10.3109/13506129.2011.574354021. Amyloid. 2011. PMID: 21838433 No abstract available.
243 results