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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.
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. 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.
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation.
Miller R, Paquette J, Barker A, Sapp E, McHugh N, Bramato B, Yamada N, Alterman J, Echeveria D, Yamada K, Watts J, Anaclet C, DiFiglia M, Khvorova A, Aronin N. Miller R, et al. bioRxiv [Preprint]. 2024 Jun 8:2024.06.06.597639. doi: 10.1101/2024.06.06.597639. bioRxiv. 2024. Update in: Mol Ther Nucleic Acids. 2024 Oct 15;35(4):102359. doi: 10.1016/j.omtn.2024.102359. PMID: 38895198 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. 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.