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Page 1
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system.
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, Khvorova A. Belgrad J, et al. Among authors: summers a. Nucleic Acids Res. 2024 Jun 24;52(11):6099-6113. doi: 10.1093/nar/gkae368. Nucleic Acids Res. 2024. PMID: 38726879 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: summers a. 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.
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: summers a. 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.
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: summers a. 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.
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: summers a. 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.
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: summers a. Nat Commun. 2023 Nov 4;14(1):7099. doi: 10.1038/s41467-023-42714-4. Nat Commun. 2023. PMID: 37925520 Free PMC article.
Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity.
Fakih HH, Tang Q, Summers A, Shin M, Buchwald JE, Gagnon R, Hariharan VN, Echeverria D, Cooper DA, Watts JK, Khvorova A, Sleiman HF. Fakih HH, et al. Among authors: summers a. Mol Ther Nucleic Acids. 2023 Nov 17;34:102080. doi: 10.1016/j.omtn.2023.102080. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38089931 Free PMC article.
A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system.
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, Khvorova A. Belgrad J, et al. Among authors: summers a. bioRxiv [Preprint]. 2023 Dec 19:2023.12.19.572404. doi: 10.1101/2023.12.19.572404. bioRxiv. 2023. Update in: Nucleic Acids Res. 2024 Jun 24;52(11):6099-6113. doi: 10.1093/nar/gkae368 PMID: 38187561 Free PMC article. Updated. Preprint.
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo.
Allen S, O'Reilly D, Miller R, Sapp E, Summers A, Paquette J, Moreno DE, Bramato B, McHugh N, Yamada K, Aronin N, DiFiglia M, Khvorova A. Allen S, et al. Among authors: summers a. bioRxiv [Preprint]. 2024 Apr 28:2024.04.24.590997. doi: 10.1101/2024.04.24.590997. bioRxiv. 2024. Update in: Nucleic Acid Ther. 2024 Aug;34(4):164-172. doi: 10.1089/nat.2024.0027 PMID: 38774633 Free PMC article. Updated. Preprint.
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: summers a. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
717 results