| Exosomes produced from 3D cultures of MSCs by tangential flow filtration show higher yield and improved activity RA Haraszti, R Miller, M Stoppato, YY Sere, A Coles, MC Didiot, ... Molecular Therapy 26 (12), 2838-2847, 2018 | 553 | 2018 |
| A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system JF Alterman, BMDC Godinho, MR Hassler, CM Ferguson, D Echeverria, ... Nature biotechnology 37 (8), 884-894, 2019 | 250 | 2019 |
| Optimized cholesterol-siRNA chemistry improves productive loading onto extracellular vesicles RA Haraszti, R Miller, MC Didiot, A Biscans, JF Alterman, MR Hassler, ... Molecular Therapy 26 (8), 1973-1982, 2018 | 113 | 2018 |
| Serum deprivation of mesenchymal stem cells improves exosome activity and alters lipid and protein composition RA Haraszti, R Miller, ML Dubuke, HE Rockwell, AH Coles, E Sapp, ... Iscience 16, 230-241, 2019 | 109 | 2019 |
| 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 | 47 | 2022 |
| 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 | 40 | 2017 |
| Enhancing siRNA efficacy in vivo with extended nucleic acid backbones K Yamada, VN Hariharan, J Caiazzi, R Miller, CM Ferguson, E Sapp, ... Nature Biotechnology 43 (6), 904-913, 2025 | 34 | 2025 |
| Mutant huntingtin messenger RNA forms neuronal nuclear clusters in rodent and human brains S Ly, MC Didiot, CM Ferguson, AH Coles, R Miller, K Chase, D Echeverria, ... Brain Communications 4 (6), fcac248, 2022 | 24 | 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 | 20 | 2021 |
| Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation R Miller, J Paquette, A Barker, E Sapp, N McHugh, B Bramato, N Yamada, ... Molecular Therapy Nucleic Acids 35 (4), 2024 | 19 | 2024 |
| Extended nucleic acid (exNA): a novel, biologically compatible backbone that significantly enhances oligonucleotide efficacy in vivo K Yamada, VN Hariharan, J Caiazzi, R Miller, C Furguson, E Sapp, ... bioRxiv, 2023 | 15 | 2023 |
| 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 | 15 | 2022 |
| Divalent siRNA for prion disease JE Gentile, TL Corridon, FE Serack, D Echeverria, ZC Kennedy, ... bioRxiv, 2024.12. 05.627039, 2024 | 11 | 2024 |
| mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo S Allen, D O’Reilly, R Miller, E Sapp, A Summers, J Paquette, ... nucleic acid therapeutics 34 (4), 164-172, 2024 | 5 | 2024 |
| Nacc1 mutation in mice models rare neurodevelopmental disorder with underlying synaptic dysfunction MA Deehan, JM Kothuis, E Sapp, K Chase, Y Ke, C Seeley, M Iuliano, ... Journal of Neuroscience 44 (14), 2024 | 4 | 2024 |
| Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model HR Benatti, RD Prestigiacomo, T Taghian, R Miller, R King, MJ Gounis, ... Molecular Therapy Methods & Clinical Development 31, 2023 | 4 | 2023 |
| Detection of HTTex1p by western blot and immunostaining of HD human and mouse brain using neo-epitope antibody P90 highlights impact of CAG repeat expansion on its size … E Sapp, A Boudi, A Iwanowicz, J Belgrad, R Miller, D O’Reilly, K Yamada, ... bioRxiv, 2025 | 3 | 2025 |
| Allele-specific knockdown of mutant HTT protein via editing at coding region SNP heterozygosities S Oikemus, EL Pfister, E Sapp, KO Chase, LA Kennington, E Hudgens, ... | 2 | 2021 |
| Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation E Sapp, A Boudi, A Iwanowicz, J Belgrad, R Miller, R Robertson, ... Brain Communications 7 (5), fcaf314, 2025 | 1 | 2025 |
| Preclinical evaluation of gene therapy for neurodegenerative diseases caused by inherited deficiency of progranulin R Miller, C Hinderer, C Dyer, J Johansson, P Bell, JM Wilson HUMAN GENE THERAPY 30 (11), A91-A91, 2019 | 1 | 2019 |
Anastasia KhvorovaUMASS Medical SchoolVerified email at umassmed.edu
