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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: guenther dc. Nat Biotechnol. 2022 Oct;40(10):1500-1508. doi: 10.1038/s41587-022-01334-x. Epub 2022 Jun 2. Nat Biotechnol. 2022. PMID: 35654979
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
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: guenther dc. 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
Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5'-exonuclease.
Kumar P, Degaonkar R, Guenther DC, Abramov M, Schepers G, Capobianco M, Jiang Y, Harp J, Kaittanis C, Janas MM, Castoreno A, Zlatev I, Schlegel MK, Herdewijn P, Egli M, Manoharan M. Kumar P, et al. Among authors: guenther dc. Nucleic Acids Res. 2020 May 7;48(8):4028-4040. doi: 10.1093/nar/gkaa125. Nucleic Acids Res. 2020. PMID: 32170309 Free PMC article.
Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing.
Jahns H, Degaonkar R, Podbevsek P, Gupta S, Bisbe A, Aluri K, Szeto J, Kumar P, LeBlanc S, Racie T, Brown CR, Castoreno A, Guenther DC, Jadhav V, Maier MA, Plavec J, Egli M, Manoharan M, Zlatev I. Jahns H, et al. Among authors: guenther dc. Nucleic Acids Res. 2021 Oct 11;49(18):10250-10264. doi: 10.1093/nar/gkab724. Nucleic Acids Res. 2021. PMID: 34508350 Free PMC article.
Role of a "Magic" Methyl: 2'-Deoxy-2'-α-F-2'-β-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5'-Phosphate Mimics and Mitigation of Off-Target Effects.
Guenther DC, Mori S, Matsuda S, Gilbert JA, Willoughby JLS, Hyde S, Bisbe A, Jiang Y, Agarwal S, Madaoui M, Janas MM, Charisse K, Maier MA, Egli M, Manoharan M. Guenther DC, et al. J Am Chem Soc. 2022 Aug 17;144(32):14517-14534. doi: 10.1021/jacs.2c01679. Epub 2022 Aug 3. J Am Chem Soc. 2022. PMID: 35921401 Free PMC article.
27 results