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Year Number of Results
2015 2
2016 4
2017 7
2018 12
2019 10
2020 9
2021 6
2022 8
2023 12
2024 5

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66 results

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Page 1
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 Free PMC article.
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease.
O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, Monopoli K, Yang XW, Carroll J, DiFiglia M, Aronin N, Khvorova A. O'Reilly D, et al. Mol Ther. 2023 Jun 7;31(6):1661-1674. doi: 10.1016/j.ymthe.2023.05.006. Epub 2023 May 12. Mol Ther. 2023. PMID: 37177784 Free article.
RNAi-based modulation of IFN-γ signaling in skin.
Tang Q, Sousa J, Echeverria D, Fan X, Hsueh YC, Afshari K, MeHugh N, Cooper DA, Vangjeli L, Monopoli K, Okamura K, Biscans A, Clauss A, Harris JE, Khvorova A. Tang Q, et al. Mol Ther. 2022 Aug 3;30(8):2709-2721. doi: 10.1016/j.ymthe.2022.04.019. Epub 2022 Apr 27. Mol Ther. 2022. PMID: 35477658 Free PMC article.
Nucleic Acid Therapeutics for Neurological Diseases.
Watts JK, Brown RH, Khvorova A. Watts JK, et al. Neurotherapeutics. 2019 Apr;16(2):245-247. doi: 10.1007/s13311-019-00736-1. Neurotherapeutics. 2019. PMID: 31037650 Free PMC article. No abstract available.
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.
Tang Q, Fakih HH, Zain Ui Abideen M, Hildebrand SR, Afshari K, Gross KY, Sousa J, Maebius AS, Bartholdy C, Søgaard PP, Jackerott M, Hariharan V, Summers A, Fan X, Okamura K, Monopoli KR, Cooper DA, Echeverria D, Bramato B, McHugh N, Furgal RC, Dresser K, Winter SJ, Biscans A, Chuprin J, Haddadi NS, Sherman S, Yıldız-Altay Ü, Rashighi M, Richmond JM, Bouix-Peter C, Blanchard C, Clauss A, Alterman JF, Khvorova A, Harris JE. Tang Q, et al. Nat Commun. 2023 Nov 4;14(1):7099. doi: 10.1038/s41467-023-42714-4. Nat Commun. 2023. PMID: 37925520 Free PMC article.
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts.
Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodríguez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK. Shin M, et al. Nucleic Acids Res. 2022 Aug 26;50(15):8418-8430. doi: 10.1093/nar/gkac630. Nucleic Acids Res. 2022. PMID: 35920332 Free PMC article.
A combinatorial approach for achieving CNS-selective RNAi.
Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. Ferguson CM, et al. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi: 10.1093/nar/gkae100. Nucleic Acids Res. 2024. PMID: 38348876 Free PMC article.
66 results