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Page 1
Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding.
Mathews EW, Coffey SR, Gärtner A, Belgrad J, Bragg RM, O'Reilly D, Cantle JP, McHugh C, Summers A, Fentz J, Schwagarus T, Cornelius A, Lingos I, Burch Z, Kovalenko M, Andrew MA, Frank Bennett C, Kordasiewicz HB, Marchionini DM, Wilkinson H, Vogt TF, Pinto RM, Khvorova A, Howland D, Wheeler VC, Carroll JB. Mathews EW, et al. Among authors: khvorova a. bioRxiv [Preprint]. 2024 Nov 4:2024.11.04.619693. doi: 10.1101/2024.11.04.619693. bioRxiv. 2024. PMID: 39574582 Free PMC article. 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: khvorova 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.
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: khvorova 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.
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.
Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. Engelbeen S, et al. Among authors: khvorova a. Nucleic Acid Ther. 2023 Dec;33(6):348-360. doi: 10.1089/nat.2023.0038. Nucleic Acid Ther. 2023. PMID: 38010230 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: khvorova 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.
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
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model.
Benatti HR, Prestigiacomo RD, Taghian T, Miller R, King R, Gounis MJ, Celik U, Bertrand S, Tuominen S, Bierfeldt L, Parsley E, Gallagher J, Hall EF, McElroy AW, Sena-Esteves M, Khvorova A, Aronin N, Gray-Edwards HL. Benatti HR, et al. Among authors: khvorova a. Mol Ther Methods Clin Dev. 2023 Sep 26;31:101122. doi: 10.1016/j.omtm.2023.101122. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 37920238 Free PMC article.
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. Among authors: khvorova a. Mol Ther Nucleic Acids. 2024 Oct 15;35(4):102359. doi: 10.1016/j.omtn.2024.102359. eCollection 2024 Dec 10. Mol Ther Nucleic Acids. 2024. PMID: 39554992 Free PMC article.
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: khvorova a. 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.
154 results