| 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 | 357 | 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 | 154 | 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 | 82 | 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 | 74 | 2019 |
| Hydrophobicity of lipid-conjugated siRNAs predicts productive loading to small extracellular vesicles A Biscans, RA Haraszti, D Echeverria, R Miller, MC Didiot, M Nikan, ... Molecular Therapy 26 (6), 1520-1528, 2018 | 39 | 2018 |
| 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 |
| 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 | 13 | 2022 |
| 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 | 9 | 2022 |
| 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 | 7 | 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 |
| 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 | 5 | 2023 |
| 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 |
| Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo VN Hariharan, J Caiazzi, R Miller, C Ferguson, E Sapp, H Fakih, Q Tang, ... Research Square, 2023 | 1 | 2023 |
| 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 | | 2024 |
| mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo SJ Allen, D O'Reilly, R Miller, E Sapp, A Summers, J Paquette, DE Moreno, ... bioRxiv, 2024.04. 24.590997, 2024 | | 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 | | 2023 |
| Rapalog-Mediated Regulation of an AAV miRNA Targeting Huntingtin in a HD Transgenic Mouse Model R Miller, E Pfister, E Sapp, M DiFiglia, N Aronin, C Mueller MOLECULAR THERAPY 28 (4), 70-71, 2020 | | 2020 |
| Engineered exosomes for non-viral delivery of RNA therapeutics to brain RA Haraszti, MC Didiot, A Biscans, R Miller, M Dubuke, H Rockwell, ... HUMAN GENE THERAPY 28 (12), A16-A16, 2017 | | 2017 |
| ARRDC1-and Histone-Enriched Exosomes Released from Starved Stem Cells Improve siRNA Transfer to Neurons RA Haraszti, R Miller, MC Didiot, M Dubuke, J Leszyk, SA Shaffer, ... MOLECULAR THERAPY 25 (5), 218-219, 2017 | | 2017 |
