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An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis.
Yenilmez B, Wetoska N, Kelly M, Echeverria D, Min K, Lifshitz L, Alterman JF, Hassler MR, Hildebrand S, DiMarzio C, McHugh N, Vangjeli L, Sousa J, Pan M, Han X, Brehm MA, Khvorova A, Czech MP. Yenilmez B, et al. Among authors: khvorova a. Mol Ther. 2022 Mar 2;30(3):1329-1342. doi: 10.1016/j.ymthe.2021.11.007. Epub 2021 Nov 11. Mol Ther. 2022. PMID: 34774753 Free PMC 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.
Visualization of self-delivering hydrophobically modified siRNA cellular internalization.
Ly S, Navaroli DM, Didiot MC, Cardia J, Pandarinathan L, Alterman JF, Fogarty K, Standley C, Lifshitz LM, Bellve KD, Prot M, Echeverria D, Corvera S, Khvorova A. Ly S, et al. Among authors: khvorova a. Nucleic Acids Res. 2017 Jan 9;45(1):15-25. doi: 10.1093/nar/gkw1005. Epub 2016 Nov 28. Nucleic Acids Res. 2017. PMID: 27899655 Free PMC article.
153 results