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Page 1
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones.
Yamada K, Hariharan VN, Caiazzi J, Miller R, Ferguson CM, Sapp E, Fakih HH, Tang Q, Yamada N, Furgal RC, Paquette JD, Biscans A, Bramato BM, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Jackson SO, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: furgal rc. Nat Biotechnol. 2024 Aug 1. doi: 10.1038/s41587-024-02336-7. Online ahead of print. Nat Biotechnol. 2024. PMID: 39090305
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. Among authors: furgal r. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
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. Among authors: furgal rc. Nat Commun. 2023 Nov 4;14(1):7099. doi: 10.1038/s41467-023-42714-4. Nat Commun. 2023. PMID: 37925520 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. Among authors: furgal r. Mol Ther. 2023 Nov 1;31(11):3355-3356. doi: 10.1016/j.ymthe.2023.09.016. Epub 2023 Sep 25. Mol Ther. 2023. PMID: 37751745 Free PMC article. No abstract available.
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Hariharan VN, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, Tang Q, Yamada N, Furgal R, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho B, Hildebrand S, Echeverria D, Hassler M, Alterman J, DiFiglia M, Aronin N, Khvorova A, Yamada K. Hariharan VN, et al. Among authors: furgal r. Res Sq [Preprint]. 2023 Jun 1:rs.3.rs-2987323. doi: 10.21203/rs.3.rs-2987323/v1. Res Sq. 2023. Update in: Nat Biotechnol. 2024 Aug 1. doi: 10.1038/s41587-024-02336-7 PMID: 37398145 Free PMC article. Updated. Preprint.
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Yamada K, Hariharan VN, Caiazzi J, Miller R, Furguson C, Sapp E, Fakih H, Tan Q, Yamada N, Furgal RC, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: furgal rc. bioRxiv [Preprint]. 2023 May 26:2023.05.26.542506. doi: 10.1101/2023.05.26.542506. bioRxiv. 2023. Update in: Nat Biotechnol. 2024 Aug 1. doi: 10.1038/s41587-024-02336-7 PMID: 37292886 Free PMC article. Updated. Preprint.
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. Among authors: furgal r. 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.