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 |
Hydrophobically modified siRNAs silence huntingtin mRNA in primary neurons and mouse brain JF Alterman, LM Hall, AH Coles, MR Hassler, MC Didiot, K Chase, ... Molecular Therapy-Nucleic Acids 4, 2015 | 147 | 2015 |
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 |
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 |
Fully stabilized asymmetric sirna A Khvorova, N Aronin, M Hassler, J Alterman US Patent App. 15/089,423, 2016 | 67 | 2016 |
Oligonucleotide compounds for targeting huntingtin mRNA A Khvorova, N Aronin, J Alterman US Patent 9,809,817, 2017 | 64 | 2017 |
Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs MF Osborn, JF Alterman, M Nikan, H Cao, MC Didiot, MR Hassler, ... Nucleic acids research 43 (18), 8664-8672, 2015 | 50 | 2015 |
A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo AH Coles, MF Osborn, JF Alterman, AA Turanov, BMDC Godinho, ... nucleic acid therapeutics 26 (2), 86-92, 2016 | 42 | 2016 |
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 |
Synthesis and biological properties of triazole-linked locked nucleic acid VK Sharma, SK Singh, PM Krishnamurthy, JF Alterman, RA Haraszti, ... Chemical communications 53 (63), 8906-8909, 2017 | 36 | 2017 |
Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease RL McAdam, A Morton, SL Gordon, JF Alterman, A Khvorova, MA Cousin, ... Neurobiology of disease 134, 104637, 2020 | 28 | 2020 |
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis B Yenilmez, N Wetoska, M Kelly, D Echeverria, K Min, L Lifshitz, ... Molecular Therapy 30 (3), 1329-1342, 2022 | 22 | 2022 |
Branched oligonucleotides A Khvorova, M Hassler, J Alterman, BM da Cruz Godinho US Patent 10,478,503, 2019 | 21 | 2019 |
Fully stabilized asymmetric sirna A Khvorova, N Aronin, M Hassler, J Alterman US Patent App. 15/691,120, 2017 | 20 | 2017 |