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2020 4
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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 PMC article.
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.
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin.
Tang Q, Gross KY, Fakih HH, Jackson SO, Zain U I Abideen M, Monopoli KR, Blanchard C, Bouix-Peter C, Portal T, Harris JE, Khvorova A, Alterman JF. Tang Q, et al. Mol Ther Nucleic Acids. 2024 Jan 9;35(1):102117. doi: 10.1016/j.omtn.2024.102117. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38304729 Free PMC 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.
siRNAs-A New Class of Medicines.
Khvorova A. Khvorova A. JAMA. 2023 Jun 27;329(24):2185-2186. doi: 10.1001/jama.2023.4570. JAMA. 2023. PMID: 37306980
Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.
Davis SM, Hariharan VN, Lo A, Turanov AA, Echeverria D, Sousa J, McHugh N, Biscans A, Alterman JF, Karumanchi SA, Moore MJ, Khvorova A. Davis SM, et al. Mol Ther Nucleic Acids. 2022 Jun 22;29:135-149. doi: 10.1016/j.omtn.2022.06.009. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 35847173 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.
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Weiss A, Gilbert JW, Flores IVR, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Summers A, Bramato B, McHugh N, Furgal R, Yamada N, Cooper D, Monopoli K, Godinho BMDC, Hassler MR, Yamada K, Greer P, Henninger N, Brown RH Jr, Khvorova A. Weiss A, et al. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
33 results