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Page 1
Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells.
Novobrantseva TI, Borodovsky A, Wong J, Klebanov B, Zafari M, Yucius K, Querbes W, Ge P, Ruda VM, Milstein S, Speciner L, Duncan R, Barros S, Basha G, Cullis P, Akinc A, Donahoe JS, Narayanannair Jayaprakash K, Jayaraman M, Bogorad RL, Love K, Whitehead K, Levins C, Manoharan M, Swirski FK, Weissleder R, Langer R, Anderson DG, de Fougerolles A, Nahrendorf M, Koteliansky V. Novobrantseva TI, et al. Among authors: milstein s. Mol Ther Nucleic Acids. 2012 Jan 24;1(1):e4. doi: 10.1038/mtna.2011.3. Mol Ther Nucleic Acids. 2012. PMID: 23344621 Free PMC article.
Measuring RNAi knockdown using qPCR.
Milstein S, Nguyen M, Meyers R, de Fougerolles A. Milstein S, et al. Methods Enzymol. 2013;533:57-77. doi: 10.1016/B978-0-12-420067-8.00006-4. Methods Enzymol. 2013. PMID: 24182918
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification.
Chan A, Liebow A, Yasuda M, Gan L, Racie T, Maier M, Kuchimanchi S, Foster D, Milstein S, Charisse K, Sehgal A, Manoharan M, Meyers R, Fitzgerald K, Simon A, Desnick RJ, Querbes W. Chan A, et al. Among authors: milstein s. Mol Ther Nucleic Acids. 2015 Nov 3;4(11):e263. doi: 10.1038/mtna.2015.36. Mol Ther Nucleic Acids. 2015. PMID: 26528940 Free PMC article.
Therapeutic siRNA silencing in inflammatory monocytes in mice.
Leuschner F, Dutta P, Gorbatov R, Novobrantseva TI, Donahoe JS, Courties G, Lee KM, Kim JI, Markmann JF, Marinelli B, Panizzi P, Lee WW, Iwamoto Y, Milstein S, Epstein-Barash H, Cantley W, Wong J, Cortez-Retamozo V, Newton A, Love K, Libby P, Pittet MJ, Swirski FK, Koteliansky V, Langer R, Weissleder R, Anderson DG, Nahrendorf M. Leuschner F, et al. Among authors: milstein s. Nat Biotechnol. 2011 Oct 9;29(11):1005-10. doi: 10.1038/nbt.1989. Nat Biotechnol. 2011. PMID: 21983520 Free PMC article.
Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing.
Nair JK, Willoughby JL, Chan A, Charisse K, Alam MR, Wang Q, Hoekstra M, Kandasamy P, Kel'in AV, Milstein S, Taneja N, O'Shea J, Shaikh S, Zhang L, van der Sluis RJ, Jung ME, Akinc A, Hutabarat R, Kuchimanchi S, Fitzgerald K, Zimmermann T, van Berkel TJ, Maier MA, Rajeev KG, Manoharan M. Nair JK, et al. Among authors: milstein s. J Am Chem Soc. 2014 Dec 10;136(49):16958-61. doi: 10.1021/ja505986a. Epub 2014 Dec 1. J Am Chem Soc. 2014. PMID: 25434769
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: milstein s. ACS Chem Biol. 2015 May 15;10(5):1181-7. doi: 10.1021/cb501028c. Epub 2015 Mar 2. ACS Chem Biol. 2015. PMID: 25730476
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: milstein s. Chembiochem. 2015 Apr 13;16(6):903-8. doi: 10.1002/cbic.201500023. Epub 2015 Mar 18. Chembiochem. 2015. PMID: 25786782
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
Sehgal A, Barros S, Ivanciu L, Cooley B, Qin J, Racie T, Hettinger J, Carioto M, Jiang Y, Brodsky J, Prabhala H, Zhang X, Attarwala H, Hutabarat R, Foster D, Milstein S, Charisse K, Kuchimanchi S, Maier MA, Nechev L, Kandasamy P, Kel'in AV, Nair JK, Rajeev KG, Manoharan M, Meyers R, Sorensen B, Simon AR, Dargaud Y, Negrier C, Camire RM, Akinc A. Sehgal A, et al. Among authors: milstein s. Nat Med. 2015 May;21(5):492-7. doi: 10.1038/nm.3847. Epub 2015 Apr 13. Nat Med. 2015. PMID: 25849132
Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates.
Foster DJ, Brown CR, Shaikh S, Trapp C, Schlegel MK, Qian K, Sehgal A, Rajeev KG, Jadhav V, Manoharan M, Kuchimanchi S, Maier MA, Milstein S. Foster DJ, et al. Among authors: milstein s. Mol Ther. 2018 Mar 7;26(3):708-717. doi: 10.1016/j.ymthe.2017.12.021. Epub 2018 Jan 4. Mol Ther. 2018. PMID: 29456020 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: milstein s. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 Free PMC article.
173 results