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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: 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.
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo.
Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Chen Z, Gaston N, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, Sontheimer EJ. Zhang H, et al. Among authors: khvorova a. bioRxiv [Preprint]. 2023 Mar 23:2023.03.20.533459. doi: 10.1101/2023.03.20.533459. bioRxiv. 2023. Update in: Nucleic Acids Res. 2024 Jan 25;52(2):977-997. doi: 10.1093/nar/gkad1125 PMID: 36993169 Free PMC article. Updated. Preprint.
Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis.
Min K, Yenilmez B, Kelly M, Echeverria D, Elleby M, Lifshitz LM, Raymond N, Tsagkaraki E, Harney SM, DiMarzio C, Wang H, McHugh N, Bramato B, Morrision B, Rothstein JD, Khvorova A, Czech MP. Min K, et al. Among authors: khvorova a. bioRxiv [Preprint]. 2023 Oct 19:2023.05.03.539244. doi: 10.1101/2023.05.03.539244. bioRxiv. 2023. Update in: Elife. 2024 Apr 02;12:RP89136. doi: 10.7554/eLife.89136 PMID: 37205462 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: khvorova 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.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors.
Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Cheng SY, et al. Among authors: khvorova a. bioRxiv [Preprint]. 2023 Sep 22:2023.09.20.558641. doi: 10.1101/2023.09.20.558641. bioRxiv. 2023. Update in: Mol Ther Nucleic Acids. 2023 Dec 05;35(1):102088. doi: 10.1016/j.omtn.2023.102088 PMID: 37790464 Free PMC article. Updated. Preprint.
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: khvorova 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.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.
Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Cheng SY, et al. Among authors: khvorova a. Mol Ther Nucleic Acids. 2023 Dec 5;35(1):102088. doi: 10.1016/j.omtn.2023.102088. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2023. PMID: 38192611 Free PMC article.
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin.
Tang Q, Gross KY, Fakih HH, Jackson SO, Zain U I Abideen M, Monopoli KR, Blanchard C, Bouix-Peter C, Portal T, Harris JE, Khvorova A, Alterman JF. Tang Q, et al. Among authors: khvorova a. Mol Ther Nucleic Acids. 2024 Jan 9;35(1):102117. doi: 10.1016/j.omtn.2024.102117. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38304729 Free PMC article.
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: khvorova a. 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.
155 results