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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: khvorova a. 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.
Rational siRNA design for RNA interference.
Reynolds A, Leake D, Boese Q, Scaringe S, Marshall WS, Khvorova A. Reynolds A, et al. Among authors: khvorova a. Nat Biotechnol. 2004 Mar;22(3):326-30. doi: 10.1038/nbt936. Epub 2004 Feb 1. Nat Biotechnol. 2004. PMID: 14758366 Free article.
Taking charge of siRNA delivery.
Khvorova A, Osborn MF, Hassler MR. Khvorova A, et al. 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. Nat Biotechnol. 2015 May;33(5):563. doi: 10.1038/nbt0515-563c. Nat Biotechnol. 2015. PMID: 25965761 No abstract available.
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain.
Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Alterman JF, et al. Among authors: khvorova a. Mol Ther Nucleic Acids. 2015 Dec 1;4(12):e266. doi: 10.1038/mtna.2015.38. Mol Ther Nucleic Acids. 2015. PMID: 26623938 Free PMC article.
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.
Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A. Didiot MC, et al. Among authors: khvorova a. Mol Ther. 2016 Oct;24(10):1836-1847. doi: 10.1038/mt.2016.126. Epub 2016 Jun 27. Mol Ther. 2016. PMID: 27506293 Free PMC article.
142 results