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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: miller r. 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.
Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition.
Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, Narain NR, Wollacott R, Wang Y, Shaffer SA, Kiebish MA, DiFiglia M, Aronin N, Khvorova A. Haraszti RA, et al. Among authors: miller r. iScience. 2019 Jun 28;16:230-241. doi: 10.1016/j.isci.2019.05.029. Epub 2019 May 27. iScience. 2019. PMID: 31195240 Free PMC article.
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Oikemus SR, Pfister EL, Sapp E, Chase KO, Kennington LA, Hudgens E, Miller R, Zhu LJ, Chaudhary A, Mick EO, Sena-Esteves M, Wolfe SA, DiFiglia M, Aronin N, Brodsky MH. Oikemus SR, et al. Among authors: miller r. Hum Gene Ther. 2022 Jan;33(1-2):25-36. doi: 10.1089/hum.2020.323. Hum Gene Ther. 2022. PMID: 34376056 Free PMC article.
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Yamada K, Hildebrand S, Davis SM, Miller R, Conroy F, Sapp E, Caiazzi J, Alterman JF, Roux L, Echeverria D, Hassler MR, Pfister EL, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: miller r. Nucleic Acids Res. 2021 Dec 2;49(21):12069-12088. doi: 10.1093/nar/gkab1126. Nucleic Acids Res. 2021. PMID: 34850120 Free PMC article.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, Khvorova A, Pfister EL. Conroy F, et al. Among authors: miller r. Nat Commun. 2022 Oct 3;13(1):5802. doi: 10.1038/s41467-022-33061-x. Nat Commun. 2022. PMID: 36192390 Free PMC article.
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