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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: sepp lorenzino l. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 Free PMC article.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald K, Vaishnaw AK, Smith PF, Manoharan M, Jadhav V, Wu JT, Maier MA. Janas MM, et al. Among authors: sepp lorenzino l. Nucleic Acids Res. 2019 Apr 23;47(7):3306-3320. doi: 10.1093/nar/gkz140. Nucleic Acids Res. 2019. PMID: 30820542 Free PMC article.
Centyrin ligands for extrahepatic delivery of siRNA.
Klein D, Goldberg S, Theile CS, Dambra R, Haskell K, Kuhar E, Lin T, Parmar R, Manoharan M, Richter M, Wu M, Mendrola Zarazowski J, Jadhav V, Maier MA, Sepp-Lorenzino L, O'Neil K, Dudkin V. Klein D, et al. Among authors: sepp lorenzino l. Mol Ther. 2021 Jun 2;29(6):2053-2066. doi: 10.1016/j.ymthe.2021.02.015. Epub 2021 Feb 15. Mol Ther. 2021. PMID: 33601052 Free PMC article.
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy.
Engelbeen S, Pasteuning-Vuhman S, Boertje-van der Meulen J, Parmar R, Charisse K, Sepp-Lorenzino L, Manoharan M, Aartsma-Rus A, van Putten M. Engelbeen S, et al. Among authors: sepp lorenzino l. Nucleic Acid Ther. 2023 Jan;33(1):26-34. doi: 10.1089/nat.2022.0021. Epub 2022 Oct 20. Nucleic Acid Ther. 2023. PMID: 36269327 Free PMC article.
Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth.
Khan T, Weber H, DiMuzio J, Matter A, Dogdas B, Shah T, Thankappan A, Disa J, Jadhav V, Lubbers L, Sepp-Lorenzino L, Strapps WR, Tadin-Strapps M. Khan T, et al. Mol Ther Nucleic Acids. 2016 Aug 2;5(8):e342. doi: 10.1038/mtna.2016.55. Mol Ther Nucleic Acids. 2016. PMID: 27483025 Free PMC article.
Knockdown of Virus Antigen Expression Increases Therapeutic Vaccine Efficacy in High-Titer Hepatitis B Virus Carrier Mice.
Michler T, Kosinska AD, Festag J, Bunse T, Su J, Ringelhan M, Imhof H, Grimm D, Steiger K, Mogler C, Heikenwalder M, Michel ML, Guzman CA, Milstein S, Sepp-Lorenzino L, Knolle P, Protzer U. Michler T, et al. Among authors: sepp lorenzino l. Gastroenterology. 2020 May;158(6):1762-1775.e9. doi: 10.1053/j.gastro.2020.01.032. Epub 2020 Jan 28. Gastroenterology. 2020. PMID: 32001321
83 results