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Page 1
Metabolically Stable Anomeric Linkages Containing GalNAc-siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways.
Kandasamy P, Mori S, Matsuda S, Erande N, Datta D, Willoughby JLS, Taneja N, O'Shea J, Bisbe A, Manoharan RM, Yucius K, Nguyen T, Indrakanti R, Gupta S, Gilbert JA, Racie T, Chan A, Liu J, Hutabarat R, Nair JK, Charisse K, Maier MA, Rajeev KG, Egli M, Manoharan M. Kandasamy P, et al. Among authors: manoharan rm. J Med Chem. 2023 Feb 23;66(4):2506-2523. doi: 10.1021/acs.jmedchem.2c01337. Epub 2023 Feb 9. J Med Chem. 2023. PMID: 36757090
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: manoharan rm. ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2. ACS Chem Biol. 2015. PMID: 25730476
4'-C-Methoxy-2'-deoxy-2'-fluoro Modified Ribonucleotides Improve Metabolic Stability and Elicit Efficient RNAi-Mediated Gene Silencing.
Malek-Adamian E, Guenther DC, Matsuda S, Martínez-Montero S, Zlatev I, Harp J, Burai Patrascu M, Foster DJ, Fakhoury J, Perkins L, Moitessier N, Manoharan RM, Taneja N, Bisbe A, Charisse K, Maier M, Rajeev KG, Egli M, Manoharan M, Damha MJ. Malek-Adamian E, et al. Among authors: manoharan rm. J Am Chem Soc. 2017 Oct 18;139(41):14542-14555. doi: 10.1021/jacs.7b07582. Epub 2017 Oct 6. J Am Chem Soc. 2017. PMID: 28937776
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: manoharan rm. Nucleic Acids Res. 2022 Feb 22;50(3):1221-1240. doi: 10.1093/nar/gkab544. Nucleic Acids Res. 2022. PMID: 34268578 Free PMC article.
Shorter Is Better: The α-(l)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs.
Matsuda S, Bala S, Liao JY, Datta D, Mikami A, Woods L, Harp JM, Gilbert JA, Bisbe A, Manoharan RM, Kim M, Theile CS, Guenther DC, Jiang Y, Agarwal S, Maganti R, Schlegel MK, Zlatev I, Charisse K, Rajeev KG, Castoreno A, Maier M, Janas MM, Egli M, Chaput JC, Manoharan M. Matsuda S, et al. Among authors: manoharan rm. J Am Chem Soc. 2023 Sep 13;145(36):19691-19706. doi: 10.1021/jacs.3c04744. Epub 2023 Aug 28. J Am Chem Soc. 2023. PMID: 37638886