Exosome-mediated delivery of hydrophobically modified siRNA for huntingtin mRNA silencing MC Didiot, LM Hall, AH Coles, RA Haraszti, BMDC Godinho, K Chase, ... Molecular Therapy 24 (10), 1836-1847, 2016 | 450 | 2016 |
RNAi modulation of placental sFLT1 for the treatment of preeclampsia AA Turanov, A Lo, MR Hassler, A Makris, A Ashar-Patel, JF Alterman, ... Nature biotechnology 36 (12), 1164-1173, 2018 | 159 | 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 |
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo MR Hassler, AA Turanov, JF Alterman, RA Haraszti, AH Coles, MF Osborn, ... Nucleic acids research 46 (5), 2185-2196, 2018 | 150 | 2018 |
Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo A Biscans, A Coles, R Haraszti, D Echeverria, M Hassler, M Osborn, ... Nucleic acids research 47 (3), 1082-1096, 2019 | 149 | 2019 |
Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways MF Osborn, AH Coles, A Biscans, RA Haraszti, L Roux, S Davis, S Ly, ... Nucleic acids research 47 (3), 1070-1081, 2019 | 112 | 2019 |
5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo RA Haraszti, L Roux, AH Coles, AA Turanov, JF Alterman, D Echeverria, ... Nucleic acids research 45 (13), 7581-7592, 2017 | 100 | 2017 |
Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing A Mir, JF Alterman, MR Hassler, AJ Debacker, E Hudgens, D Echeverria, ... Nature Communications 9 (1), 2641, 2018 | 96 | 2018 |
Visualization of self-delivering hydrophobically modified siRNA cellular internalization S Ly, DM Navaroli, MC Didiot, J Cardia, L Pandarinathan, JF Alterman, ... Nucleic acids research 45 (1), 15-25, 2017 | 95 | 2017 |
Docosahexaenoic acid conjugation enhances distribution and safety of siRNA upon local administration in mouse brain M Nikan, MF Osborn, AH Coles, BMDC Godinho, LM Hall, RA Haraszti, ... Molecular Therapy-Nucleic Acids 5, 2016 | 85 | 2016 |
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 | 83 | 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 |
The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo A Biscans, A Coles, D Echeverria, A Khvorova Journal of controlled release 302, 116-125, 2019 | 53 | 2019 |
Nuclear localization of huntingtin mRNA is specific to cells of neuronal origin MC Didiot, CM Ferguson, S Ly, AH Coles, AO Smith, AA Bicknell, LM Hall, ... Cell reports 24 (10), 2553-2560. e5, 2018 | 45 | 2018 |
Pharmacokinetic profiling of conjugated therapeutic oligonucleotides: a high-throughput method based upon serial blood microsampling coupled to peptide nucleic acid … BMDC Godinho, JW Gilbert, RA Haraszti, AH Coles, A Biscans, L Roux, ... nucleic acid therapeutics 27 (6), 323-334, 2017 | 44 | 2017 |
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 | 40 | 2018 |
Adaptation of the AOAC 2011.25 integrated total dietary fiber assay to determine the dietary fiber and oligosaccharide content of dry edible beans AE Kleintop, D Echeverria, LA Brick, HJ Thompson, MA Brick Journal of agricultural and food chemistry 61 (40), 9719-9726, 2013 | 38 | 2013 |
Transvascular delivery of hydrophobically modified siRNAs: gene silencing in the rat brain upon disruption of the blood-brain barrier BMDC Godinho, N Henninger, J Bouley, JF Alterman, RA Haraszti, ... Molecular Therapy 26 (11), 2580-2591, 2018 | 36 | 2018 |
Novel cluster and monomer-based GalNAc structures induce effective uptake of siRNAs in vitro and in vivo VK Sharma, MF Osborn, MR Hassler, D Echeverria, S Ly, EA Ulashchik, ... Bioconjugate chemistry 29 (7), 2478-2488, 2018 | 36 | 2018 |
Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in … M Nikan, MF Osborn, AH Coles, A Biscans, BMDC Godinho, RA Haraszti, ... Bioconjugate chemistry 28 (6), 1758-1766, 2017 | 36 | 2017 |