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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
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.
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.
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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Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N.
Pfister EL, et al. Among authors: conroy f.
Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23.
Hum Gene Ther. 2018.
PMID: 29207890
Free PMC article.
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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: conroy f.
Nucleic Acids Res. 2021 Dec 2;49(21):12069-12088. doi: 10.1093/nar/gkab1126.
Nucleic Acids Res. 2021.
PMID: 34850120
Free PMC article.
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Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin.
Pfister EL, Chase KO, Sun H, Kennington LA, Conroy F, Johnson E, Miller R, Borel F, Aronin N, Mueller C.
Pfister EL, et al. Among authors: conroy f.
Mol Ther Nucleic Acids. 2017 Jun 16;7:324-334. doi: 10.1016/j.omtn.2017.04.011. Epub 2017 Apr 14.
Mol Ther Nucleic Acids. 2017.
PMID: 28624208
Free PMC article.
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