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Page 1
Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo.
Rajeev KG, Nair JK, Jayaraman M, Charisse K, Taneja N, O'Shea J, Willoughby JL, Yucius K, Nguyen T, Shulga-Morskaya S, Milstein S, Liebow A, Querbes W, Borodovsky A, Fitzgerald K, Maier MA, Manoharan M. Rajeev KG, et al. Among authors: nguyen t. Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18. Chembiochem. 2015. PMID: 25786782
siRNA conjugates carrying sequentially assembled trivalent N-acetylgalactosamine linked through nucleosides elicit robust gene silencing in vivo in hepatocytes.
Matsuda S, Keiser K, Nair JK, Charisse K, Manoharan RM, Kretschmer P, Peng CG, V Kel'in A, Kandasamy P, Willoughby JL, Liebow A, Querbes W, Yucius K, Nguyen T, Milstein S, Maier MA, Rajeev KG, Manoharan M. Matsuda S, et al. Among authors: nguyen t. ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2. ACS Chem Biol. 2015. PMID: 25730476
Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression.
Willoughby JLS, Chan A, Sehgal A, Butler JS, Nair JK, Racie T, Shulga-Morskaya S, Nguyen T, Qian K, Yucius K, Charisse K, van Berkel TJC, Manoharan M, Rajeev KG, Maier MA, Jadhav V, Zimmermann TS. Willoughby JLS, et al. Among authors: nguyen t. Mol Ther. 2018 Jan 3;26(1):105-114. doi: 10.1016/j.ymthe.2017.08.019. Epub 2017 Sep 7. Mol Ther. 2018. PMID: 28988716 Free PMC article.
Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo.
Jahns H, Taneja N, Willoughby JLS, Akabane-Nakata M, Brown CR, Nguyen T, Bisbe A, Matsuda S, Hettinger M, Manoharan RM, Rajeev KG, Maier MA, Zlatev I, Charisse K, Egli M, Manoharan M. Jahns H, et al. Among authors: nguyen t. Nucleic Acids Res. 2022 Feb 22;50(3):1221-1240. doi: 10.1093/nar/gkab544. Nucleic Acids Res. 2022. PMID: 34268578 Free PMC article.
Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates.
Brown KM, Nair JK, Janas MM, Anglero-Rodriguez YI, Dang LTH, Peng H, Theile CS, Castellanos-Rizaldos E, Brown C, Foster D, Kurz J, Allen J, Maganti R, Li J, Matsuda S, Stricos M, Chickering T, Jung M, Wassarman K, Rollins J, Woods L, Kelin A, Guenther DC, Mobley MW, Petrulis J, McDougall R, Racie T, Bombardier J, Cha D, Agarwal S, Johnson L, Jiang Y, Lentini S, Gilbert J, Nguyen T, Chigas S, LeBlanc S, Poreci U, Kasper A, Rogers AB, Chong S, Davis W, Sutherland JE, Castoreno A, Milstein S, Schlegel MK, Zlatev I, Charisse K, Keating M, Manoharan M, Fitzgerald K, Wu JT, Maier MA, Jadhav V. Brown KM, et al. Among authors: nguyen t. Nat Biotechnol. 2022 Oct;40(10):1500-1508. doi: 10.1038/s41587-022-01334-x. Epub 2022 Jun 2. Nat Biotechnol. 2022. PMID: 35654979
Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis.
Kusner LL, Yucius K, Sengupta M, Sprague AG, Desai D, Nguyen T, Charisse K, Kuchimanchi S, Kallanthottathil R, Fitzgerald K, Kaminski HJ, Borodovsky A. Kusner LL, et al. Among authors: nguyen t. Mol Ther Methods Clin Dev. 2019 May 10;13:484-492. doi: 10.1016/j.omtm.2019.04.009. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 31193726 Free PMC article.
From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.
Schlegel MK, Janas MM, Jiang Y, Barry JD, Davis W, Agarwal S, Berman D, Brown CR, Castoreno A, LeBlanc S, Liebow A, Mayo T, Milstein S, Nguyen T, Shulga-Morskaya S, Hyde S, Schofield S, Szeto J, Woods LB, Yilmaz VO, Manoharan M, Egli M, Charissé K, Sepp-Lorenzino L, Haslett P, Fitzgerald K, Jadhav V, Maier MA. Schlegel MK, et al. Among authors: nguyen t. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 Free PMC article.
RNA interference therapeutics targeting angiotensinogen ameliorate preeclamptic phenotype in rodent models.
Haase N, Foster DJ, Cunningham MW, Bercher J, Nguyen T, Shulga-Morskaya S, Milstein S, Shaikh S, Rollins J, Golic M, Herse F, Kräker K, Bendix I, Serdar M, Napieczynska H, Heuser A, Gellhaus A, Thiele K, Wallukat G, Müller DN, LaMarca B, Dechend R. Haase N, et al. Among authors: nguyen t. J Clin Invest. 2020 Jun 1;130(6):2928-2942. doi: 10.1172/JCI99417. J Clin Invest. 2020. PMID: 32338644 Free PMC article. No abstract available.
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