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156 results

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Page 1
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.
[No title available]
[No authors listed] [No authors listed] PMID: 39418597
Advancements in APOE and dementia research: Highlights from the 2023 AAIC Advancements: APOE conference.
Kloske CM, Belloy ME, Blue EE, Bowman GR, Carrillo MC, Chen X, Chiba-Falek O, Davis AA, Paolo GD, Garretti F, Gate D, Golden LR, Heinecke JW, Herz J, Huang Y, Iadecola C, Johnson LA, Kanekiyo T, Karch CM, Khvorova A, Koppes-den Hertog SJ, Lamb BT, Lawler PE, Guen YL, Litvinchuk A, Liu CC, Mahinrad S, Marcora E, Marino C, Michaelson DM, Miller JJ, Morganti JM, Narayan PS, Naslavsky MS, Oosthoek M, Ramachandran KV, Ramakrishnan A, Raulin AC, Robert A, Saleh RNM, Sexton C, Shah N, Shue F, Sible IJ, Soranno A, Strickland MR, Tcw J, Thierry M, Tsai LH, Tuckey RA, Ulrich JD, van der Kant R, Wang N, Wellington CL, Weninger SC, Yassine HN, Zhao N, Bu G, Goate AM, Holtzman DM. Kloske CM, et al. Among authors: khvorova a. Alzheimers Dement. 2024 Sep;20(9):6590-6605. doi: 10.1002/alz.13877. Epub 2024 Jul 19. Alzheimers Dement. 2024. PMID: 39031528 Free PMC article. Review.
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.
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.
156 results