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2007 1
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40 results

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Page 1
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Among authors: hassler mr. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 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: hassler mr. Nat Biotechnol. 2025 Jun;43(6):904-913. doi: 10.1038/s41587-024-02336-7. Epub 2024 Aug 1. Nat Biotechnol. 2025. PMID: 39090305
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice.
Weiss A, Gilbert JW, Rivera Flores IV, Belgrad J, Ferguson C, Dogan EO, Wightman N, Mocarski K, Echeverria D, Harkins AL, 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: hassler mr. Mol Ther. 2025 Aug 6;33(8):3917-3938. doi: 10.1016/j.ymthe.2025.05.010. Epub 2025 May 9. Mol Ther. 2025. PMID: 40349108 Free 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: hassler m. 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.
Divalent siRNA for prion disease.
Gentile JE, Corridon TL, Serack FE, Echeverria D, Kennedy ZC, Gallant-Behm CL, Hassler MR, Kinberger GA, Kelemen MN, Kamath NG, Lian Y, Gross KY, Miller R, DeSouza-Lenz K, Howard M, Guzman K, Chan N, Laversenne V, Curtis DT, Fettes K, Lemaitre M, Jackson AL, Yamada K, Alterman JF, Coffey AA, Minikel EV, Khvorova A, Vallabh SM. Gentile JE, et al. Among authors: hassler mr. bioRxiv [Preprint]. 2026 Mar 5:2024.12.05.627039. doi: 10.1101/2024.12.05.627039. bioRxiv. 2026. Update in: Nucleic Acids Res. 2026 Apr 23;54(8):gkag287. doi: 10.1093/nar/gkag287. PMID: 41867745 Free PMC article. Updated. Preprint.
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: hassler m. Nucleic Acids Res. 2024 May 22;52(9):5273-5284. doi: 10.1093/nar/gkae100. Nucleic Acids Res. 2024. PMID: 38348876 Free PMC article.
Divalent siRNA for prion disease.
Gentile JE, Corridon TL, Serack FE, Echeverria D, Kennedy ZC, Gallant-Behm CL, Hassler MR, Kinberger GA, Kelemen MN, Kamath NG, Lian Y, Gross KY, Miller R, DeSouza-Lenz K, Howard M, Guzman K, Chan N, Laversenne V, Curtis D, Fettes K, Lemaitre M, Jackson AL, Yamada K, Alterman JF, Coffey AA, Minikel EV, Khvorova A, Vallabh SM. Gentile JE, et al. Among authors: hassler mr. Nucleic Acids Res. 2026 Apr 23;54(8):gkag287. doi: 10.1093/nar/gkag287. Nucleic Acids Res. 2026. PMID: 42033217 Free PMC article.
Taking charge of siRNA delivery.
Khvorova A, Osborn MF, Hassler MR. Khvorova A, et al. Among authors: hassler mr. Nat Biotechnol. 2014 Dec;32(12):1197-8. doi: 10.1038/nbt.3091. Nat Biotechnol. 2014. PMID: 25489835
Corrigendum: Taking charge of siRNA delivery.
Khvorova A, Osborn MF, Hassler MR. Khvorova A, et al. Among authors: hassler mr. Nat Biotechnol. 2015 May;33(5):563. doi: 10.1038/nbt0515-563c. Nat Biotechnol. 2015. PMID: 25965761 No abstract available.
40 results