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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. Nucleic Acids Res. 2021 Oct 11;49(18):10250-10264. doi: 10.1093/nar/gkab724. Nucleic Acids Res. 2021. PMID: 34508350 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: castoreno a. Nat Biotechnol. 2022 Oct;40(10):1500-1508. doi: 10.1038/s41587-022-01334-x. Epub 2022 Jun 2. Nat Biotechnol. 2022. PMID: 35654979
Reversal of siRNA-mediated gene silencing in vivo.
Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, Schlegel MK, Degaonkar R, Shulga-Morskaya S, Xu H, Gupta S, Matsuda S, Akinc A, Rajeev KG, Manoharan M, Maier MA, Jadhav V. Zlatev I, et al. Nat Biotechnol. 2018 Jul;36(6):509-511. doi: 10.1038/nbt.4136. Epub 2018 May 14. Nat Biotechnol. 2018. PMID: 29786096
RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes.
Subramanian M, McIninch J, Zlatev I, Schlegel MK, Kaittanis C, Nguyen T, Agarwal S, Racie T, Alvarado MA, Wassarman K, Collins TS, Chickering T, Brown CR, Schmidt K, Castoreno AB, Shulga-Morskaya S, Stamenova E, Buckowing K, Berman D, Barry JD, Bisbe A, Maier MA, Fitzgerald K, Jadhav V. Subramanian M, et al. Among authors: castoreno ab. Nat Commun. 2023 Apr 8;14(1):1970. doi: 10.1038/s41467-023-37774-5. Nat Commun. 2023. PMID: 37031257 Free PMC article.
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. Nucleic Acids Res. 2020 May 7;48(8):4028-4040. doi: 10.1093/nar/gkaa125. Nucleic Acids Res. 2020. PMID: 32170309 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. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 Free PMC article.
27 results