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Page 1
Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs.
Gilbert JW, Kennedy Z, Godinho BMDC, Summers A, Weiss A, Echeverria D, Bramato B, McHugh N, Cooper D, Yamada K, Hassler M, Tran H, Gao FB, Brown RH Jr, Khvorova A. Gilbert JW, et al. Among authors: godinho bmdc. Mol Ther Nucleic Acids. 2024 Jul 31;35(3):102291. doi: 10.1016/j.omtn.2024.102291. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 39233852 Free PMC article.
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: godinho bmdc. 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: godinho bmdc. bioRxiv [Preprint]. 2024 Jun 25:2024.06.20.599943. doi: 10.1101/2024.06.20.599943. bioRxiv. 2024. PMID: 38979291 Free PMC article. Preprint.
Silencing Apoe with divalent-siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease.
Ferguson CM, Hildebrand S, Godinho BMDC, Buchwald J, Echeverria D, Coles A, Grigorenko A, Vangjeli L, Sousa J, McHugh N, Hassler M, Santarelli F, Heneka MT, Rogaev E, Khvorova A. Ferguson CM, et al. Among authors: godinho bmdc. Alzheimers Dement. 2024 Apr;20(4):2632-2652. doi: 10.1002/alz.13703. Epub 2024 Feb 20. Alzheimers Dement. 2024. PMID: 38375983 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. Among authors: godinho bmdc. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi: 10.1093/nar/gkae100. Nucleic Acids Res. 2024. PMID: 38348876 Free PMC article.
Dose-dependent reduction of somatic expansions but not Htt aggregates by di-valent siRNA-mediated silencing of MSH3 in HdhQ111 mice.
Driscoll R, Hampton L, Abraham NA, Larigan JD, Joseph NF, Hernandez-Vega JC, Geisler S, Yang FC, Deninger M, Tran DT, Khatri N, Godinho BMDC, Kinberger GA, Montagna DR, Hirst WD, Guardado CL, Glajch KE, Arnold HM, Gallant-Behm CL, Weihofen A. Driscoll R, et al. Among authors: godinho bmdc. Sci Rep. 2024 Jan 24;14(1):2061. doi: 10.1038/s41598-024-52667-3. Sci Rep. 2024. PMID: 38267530 Free PMC article.
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: godinho b. 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: godinho bmdc. 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.
40 results