| Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits M DiFiglia, M Sena-Esteves, K Chase, E Sapp, E Pfister, M Sass, J Yoder, ... Proceedings of the National Academy of Sciences 104 (43), 17204-17209, 2007 | 515 | 2007 |
| Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients EL Pfister, L Kennington, J Straubhaar, S Wagh, W Liu, M DiFiglia, ... Current Biology 19 (9), 774-778, 2009 | 303 | 2009 |
| HTT-lowering reverses Huntington’s disease immune dysfunction caused by NFκB pathway dysregulation U Träger, R Andre, N Lahiri, A Magnusson-Lind, A Weiss, S Grueninger, ... Brain 137 (3), 819-833, 2014 | 176 | 2014 |
| Artificial miRNAs reduce human mutant Huntingtin throughout the striatum in a transgenic sheep model of Huntington's disease EL Pfister, N DiNardo, E Mondo, F Borel, F Conroy, C Fraser, G Gernoux, ... Human gene therapy 29 (6), 663-673, 2018 | 99 | 2018 |
| Alterations in mRNA 3′ UTR isoform abundance accompany gene expression changes in human Huntington’s disease brains L Romo, A Ashar-Patel, E Pfister, N Aronin Cell reports 20 (13), 3057-3070, 2017 | 44 | 2017 |
| Cellular analysis of silencing the Huntington’s disease gene using AAV9 mediated delivery of artificial micro RNA into the striatum of Q140/Q140 mice AM Keeler, E Sapp, K Chase, E Sottosanti, E Danielson, E Pfister, ... Journal of Huntington's disease 5 (3), 239-248, 2016 | 37 | 2016 |
| Increased steady-state mutant huntingtin mRNA in Huntington's disease brain W Liu, J Chaurette, EL Pfister, LA Kennington, KO Chase, J Bullock, ... Journal of Huntington's Disease 2 (4), 491-500, 2013 | 34 | 2013 |
| Safe and efficient silencing with a Pol II, but not a Pol lII, promoter expressing an artificial miRNA targeting human huntingtin EL Pfister, KO Chase, H Sun, LA Kennington, F Conroy, E Johnson, ... Molecular Therapy-Nucleic Acids 7, 324-334, 2017 | 33 | 2017 |
| Huntington’s disease: silencing a brutal killer EL Pfister, PD Zamore Experimental neurology 220 (2), 226, 2009 | 32 | 2009 |
| Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease N Aronin, E Pfister, PD Zamore US Patent 8,987,222, 2015 | 31 | 2015 |
| Allele-selective suppression of mutant Huntingtin in primary human blood cells JRC Miller, EL Pfister, W Liu, R Andre, U Träger, LA Kennington, K Lo, ... Scientific reports 7 (1), 46740, 2017 | 25 | 2017 |
| Selective neuronal uptake and distribution of AAVrh8, AAV9, and AAVrh10 in sheep after intra-striatal administration E Mondo, R Moser, G Gao, C Mueller, M Sena-Esteves, E Sapp, E Pfister, ... Journal of Huntington's Disease 7 (4), 309-319, 2018 | 18 | 2018 |
| Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models F Conroy, R Miller, JF Alterman, MR Hassler, D Echeverria, ... Nature Communications 13 (1), 5802, 2022 | 12 | 2022 |
| Safety of striatal infusion of siRNA in a transgenic Huntington’s disease mouse model E Johnson, K Chase, S McGowan, E Mondo, E Pfister, E Mick, ... Journal of Huntington's disease 4 (3), 219-229, 2015 | 12 | 2015 |
| Allele-specific knockdown of mutant huntingtin protein via editing at coding region single nucleotide polymorphism heterozygosities SR Oikemus, EL Pfister, E Sapp, KO Chase, LA Kennington, E Hudgens, ... Human Gene Therapy 33 (1-2), 25-36, 2022 | 9* | 2022 |
| Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity K Yamada, S Hildebrand, SM Davis, R Miller, F Conroy, E Sapp, J Caiazzi, ... Nucleic acids research 49 (21), 12069-12088, 2021 | 7 | 2021 |
| AAV treatment of Huntington's disease C Mueller, N Aronin, EL Pfister US Patent 10,457,940, 2019 | 6 | 2019 |
| Does the mutant CAG expansion in huntingtin mRNA interfere with exonucleolytic cleavage of its first exon? W Liu, EL Pfister, LA Kennington, KO Chase, C Mueller, M DiFiglia, ... Journal of Huntington's disease 5 (1), 33-38, 2016 | 4 | 2016 |
| Toward a comprehensive catalog of regulatory elements K Fan, E Pfister, Z Weng Human Genetics 142 (8), 1091-1111, 2023 | 3 | 2023 |
| Huntington’s Disease: Les Jeux Sont Faits? EL Pfister, N Aronin Trends in Molecular Medicine 26 (10), 889-890, 2020 | 3 | 2020 |
