| Year |
Citation |
Score |
| 2024 |
Gilbert JW, Kennedy Z, Godinho BMDC, Summers A, Weiss A, Echeverria D, Bramato B, McHugh N, Cooper D, Yamada K, Hassler M, Tran H, Gao FB, Brown RH, Khvorova A. Identification of selective and non-selective targeting active siRNAs. Molecular Therapy. Nucleic Acids. 35: 102291. PMID 39233852 DOI: 10.1016/j.omtn.2024.102291 |
0.304 |
|
| 2024 |
Rivera Flores IV, Monopoli K, Jackson S, Echeverria D, O'Reilly D, Brown RH, Khvorova A. Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy. Nucleic Acid Therapeutics. PMID 39189114 DOI: 10.1089/nat.2024.0030 |
0.686 |
|
| 2024 |
Yamada K, Hariharan VN, Caiazzi J, Miller R, Ferguson CM, Sapp E, Fakih HH, Tang Q, Yamada N, Furgal RC, Paquette JD, Biscans A, Bramato BM, McHugh N, Summers A, ... ... Khvorova A, et al. Enhancing siRNA efficacy in vivo with extended nucleic acid backbones. Nature Biotechnology. PMID 39090305 DOI: 10.1038/s41587-024-02336-7 |
0.365 |
|
| 2024 |
Allen S, O'Reilly D, Miller R, Sapp E, Summers A, Paquette J, Echeverria Moreno D, Bramato B, McHugh N, Yamada K, Aronin N, DiFiglia M, Khvorova A. mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs . Nucleic Acid Therapeutics. PMID 39023561 DOI: 10.1089/nat.2024.0027 |
0.693 |
|
| 2024 |
Allen S, O'Reilly D, Miller R, Sapp E, Summers A, Paquette J, Moreno DE, Bramato B, McHugh N, Yamada K, Aronin N, DiFiglia M, Khvorova A. mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs . Biorxiv : the Preprint Server For Biology. PMID 38774633 DOI: 10.1101/2024.04.24.590997 |
0.693 |
|
| 2024 |
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, ... Khvorova A, et al. A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic Acids Research. PMID 38726879 DOI: 10.1093/nar/gkae368 |
0.307 |
|
| 2024 |
Sarli SL, Fakih HH, Kelly K, Devi G, Rembetsy-Brown JM, McEachern HR, Ferguson CM, Echeverria D, Lee J, Sousa J, Sleiman HF, Khvorova A, Watts JK. Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Research. PMID 38613388 DOI: 10.1093/nar/gkae260 |
0.323 |
|
| 2024 |
Ferguson CM, Godinho BMDC, Echeverria D, Hassler M, Vangjeli L, Sousa J, McHugh N, Alterman J, Hariharan V, Krishnamurthy PM, Watts J, Rogaev E, Khvorova A. A combinatorial approach for achieving CNS-selective RNAi. Nucleic Acids Research. PMID 38348876 DOI: 10.1093/nar/gkae100 |
0.323 |
|
| 2023 |
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, ... Khvorova A, et al. A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system. Biorxiv : the Preprint Server For Biology. PMID 38187561 DOI: 10.1101/2023.12.19.572404 |
0.302 |
|
| 2023 |
Fakih HH, Tang Q, Summers A, Shin M, Buchwald JE, Gagnon R, Hariharan VN, Echeverria D, Cooper DA, Watts JK, Khvorova A, Sleiman HF. Dendritic amphiphilic siRNA: Selective albumin binding, efficacy, and low toxicity. Molecular Therapy. Nucleic Acids. 34: 102080. PMID 38089931 DOI: 10.1016/j.omtn.2023.102080 |
0.313 |
|
| 2023 |
Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Amrani N, Chen Z, Gaston N, Wagh A, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, ... Khvorova A, et al. Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo. Nucleic Acids Research. PMID 38033325 DOI: 10.1093/nar/gkad1125 |
0.361 |
|
| 2023 |
Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. Challenges of Assessing Exon 53 Skipping of the Human Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic Acid Therapeutics. 33: 348-360. PMID 38010230 DOI: 10.1089/nat.2023.0038 |
0.756 |
|
| 2023 |
O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, ... ... Khvorova A, et al. Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 37751745 DOI: 10.1016/j.ymthe.2023.09.016 |
0.652 |
|
| 2023 |
Hariharan VN, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, Tang Q, Yamada N, Furgal R, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho B, ... ... Khvorova A, et al. Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo. Research Square. PMID 37398145 DOI: 10.21203/rs.3.rs-2987323/v1 |
0.76 |
|
| 2023 |
Yamada K, Hariharan VN, Caiazzi J, Miller R, Furguson C, Sapp E, Fakih H, Tan Q, Yamada N, Furgal RC, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, ... ... Khvorova A, et al. Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy . Biorxiv : the Preprint Server For Biology. PMID 37292886 DOI: 10.1101/2023.05.26.542506 |
0.324 |
|
| 2023 |
O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, ... ... Khvorova A, et al. Di-valent siRNA Mediated Silencing of MSH3 Blocks Somatic Repeat Expansion in Mouse Models of Huntington's Disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 37177784 DOI: 10.1016/j.ymthe.2023.05.006 |
0.702 |
|
| 2023 |
Zhang H, Kelly K, Lee J, Echeverria D, Cooper D, Panwala R, Chen Z, Gaston N, Newby GA, Xie J, Liu DR, Gao G, Wolfe SA, Khvorova A, Watts JK, et al. Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing . Biorxiv : the Preprint Server For Biology. PMID 36993169 DOI: 10.1101/2023.03.20.533459 |
0.352 |
|
| 2023 |
Hariharan VN, Shin M, Chang CW, O'Reilly D, Biscans A, Yamada K, Guo Z, Somasundaran M, Tang Q, Monopoli K, Krishnamurthy PM, Devi G, McHugh N, Cooper DA, Echeverria D, ... ... Khvorova A, et al. Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection. Proceedings of the National Academy of Sciences of the United States of America. 120: e2219523120. PMID 36893269 DOI: 10.1073/pnas.2219523120 |
0.674 |
|
| 2022 |
Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, ... Khvorova A, et al. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models. Nature Communications. 13: 5802. PMID 36192390 DOI: 10.1038/s41467-022-33061-x |
0.32 |
|
| 2022 |
Biscans A, Ly S, McHugh N, Cooper DA, Khvorova A. Engineered ionizable lipid siRNA conjugates enhance endosomal escape but induce toxicity in vivo. Journal of Controlled Release : Official Journal of the Controlled Release Society. 349: 831-843. PMID 35917865 DOI: 10.1016/j.jconrel.2022.07.041 |
0.351 |
|
| 2022 |
Davis SM, Hariharan VN, Lo A, Turanov AA, Echeverria D, Sousa J, McHugh N, Biscans A, Alterman JF, Karumanchi SA, Moore MJ, Khvorova A. Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1. Molecular Therapy. Nucleic Acids. 29: 135-149. PMID 35847173 DOI: 10.1016/j.omtn.2022.06.009 |
0.332 |
|
| 2021 |
Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, ... ... Khvorova A, et al. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. Jci Insight. 6. PMID 34935646 DOI: 10.1172/jci.insight.152203 |
0.744 |
|
| 2020 |
Ly S, Echeverria D, Sousa J, Khvorova A. Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy. Molecular Therapy. Nucleic Acids. 21: 991-1005. PMID 32818923 DOI: 10.1016/J.Omtn.2020.07.029 |
0.476 |
|
| 2020 |
Biscans A, Caiazzi J, Davis S, McHugh N, Sousa J, Khvorova A. The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy. Nucleic Acids Research. PMID 32672813 DOI: 10.1093/Nar/Gkaa595 |
0.417 |
|
| 2020 |
Ferguson CM, Echeverria D, Hassler M, Ly S, Khvorova A. Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference. Molecular Therapy. Nucleic Acids. 21: 384-393. PMID 32650236 DOI: 10.1016/J.Omtn.2020.06.006 |
0.4 |
|
| 2020 |
Davis SM, Sousa J, Vangjeli L, Hassler MR, Echeverria D, Knox E, Turanov AA, Alterman JF, Khvorova A. 2'-O-Methyl at 20-mer Guide Strand 3' Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA. Molecular Therapy. Nucleic Acids. 21: 266-277. PMID 32610253 DOI: 10.1016/J.Omtn.2020.05.010 |
0.383 |
|
| 2020 |
Sava V, Fihurka O, Khvorova A, Sanchez-Ramos J. Data on enrichment of chitosan nanoparticles for intranasal delivery of oligonucleotides to the brain. Data in Brief. 28: 105093. PMID 31970274 DOI: 10.1016/J.Dib.2019.105093 |
0.315 |
|
| 2019 |
Segal M, Biscans A, Gilles ME, Anastasiadou E, De Luca R, Lim J, Khvorova A, Slack FJ. Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 In Vivo. Molecular Therapy. Nucleic Acids. 19: 267-277. PMID 31855835 DOI: 10.1016/J.Omtn.2019.11.008 |
0.315 |
|
| 2019 |
Sava V, Fihurka O, Khvorova A, Sanchez-Ramos J. Enriched chitosan nanoparticles loaded with siRNA are effective in lowering Huntington's disease gene expression following intranasal administration. Nanomedicine : Nanotechnology, Biology, and Medicine. 102119. PMID 31666200 DOI: 10.1016/J.Nano.2019.102119 |
0.417 |
|
| 2019 |
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, ... ... Khvorova A, et al. A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system. Nature Biotechnology. 37: 884-894. PMID 31375812 DOI: 10.1038/S41587-019-0205-0 |
0.776 |
|
| 2019 |
Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, ... ... Khvorova A, et al. Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition. Iscience. 16: 230-241. PMID 31195240 DOI: 10.1016/J.Isci.2019.05.029 |
0.756 |
|
| 2019 |
Biscans A, Coles A, Echeverria D, Khvorova A. The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo. Journal of Controlled Release : Official Journal of the Controlled Release Society. 302: 116-125. PMID 30940496 DOI: 10.1016/J.Jconrel.2019.03.028 |
0.435 |
|
| 2019 |
Thijssen MF, Brüggenwirth IMA, Gillooly A, Khvorova A, Kowalik TF, Martins PN. Gene Silencing With siRNA (RNA Interference): A New Therapeutic Option During Ex Vivo Machine Liver Perfusion Preservation Liver Transplantation : Official Publication of the American Association For the Study of Liver Diseases and the International Liver Transplantation Society. 25: 140-151. PMID 30561891 DOI: 10.1002/Lt.25383 |
0.379 |
|
| 2019 |
Biscans A, Coles A, Haraszti R, Echeverria D, Hassler M, Osborn M, Khvorova A. Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo. Nucleic Acids Research. 47: 1082-1096. PMID 30544191 DOI: 10.1093/Nar/Gky1239 |
0.761 |
|
| 2019 |
Ferguson C, Grigorenko A, Alterman J, Hassler M, Coles A, Echeverria D, Rogaev E, Khvorova A. P3-063: Targeting Apolipoprotein E With Tissue Specific Small Interfering Rnas Alzheimers & Dementia. 15. DOI: 10.1016/J.Jalz.2019.06.3090 |
0.387 |
|
| 2018 |
Théry C, Witwer KW, Aikawa E, Alcaraz MJ, Anderson JD, Andriantsitohaina R, Antoniou A, Arab T, Archer F, Atkin-Smith GK, Ayre DC, Bach JM, Bachurski D, Baharvand H, Balaj L, ... ... Khvorova A, et al. Minimal information for studies of extracellular vesicles 2018 (MISEV2018): a position statement of the International Society for Extracellular Vesicles and update of the MISEV2014 guidelines. Journal of Extracellular Vesicles. 7: 1535750. PMID 30637094 DOI: 10.1080/20013078.2018.1535750 |
0.707 |
|
| 2018 |
Tousley A, Iuliano M, Weisman E, Sapp E, Zhang N, Vodicka P, Alexander J, Aviolat H, Gatune L, Reeves P, Li X, Khvorova A, Ellerby ML, Aronin N, DiFiglia M, et al. Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington's Disease. Journal of Huntington's Disease. PMID 30594931 DOI: 10.3233/Jhd-180311 |
0.301 |
|
| 2018 |
Osborn MF, Coles AH, Biscans A, Haraszti RA, Roux L, Davis S, Ly S, Echeverria D, Hassler MR, Godinho BMDC, Nikan M, Khvorova A. Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways. Nucleic Acids Research. PMID 30535404 DOI: 10.1093/Nar/Gky1232 |
0.752 |
|
| 2018 |
Turanov AA, Lo A, Hassler MR, Makris A, Ashar-Patel A, Alterman JF, Coles AH, Haraszti RA, Roux L, Godinho BMDC, Echeverria D, Pears S, Iliopoulos J, Shanmugalingam R, Ogle R, ... ... Khvorova A, et al. RNAi modulation of placental sFLT1 for the treatment of preeclampsia. Nature Biotechnology. PMID 30451990 DOI: 10.1038/Nbt.4297 |
0.739 |
|
| 2018 |
Haraszti RA, Miller R, Stoppato M, Sere YY, Coles A, Didiot MC, Wollacott R, Sapp E, Dubuke M, Li X, Shaffer S, DiFiglia M, Wang Y, Aronin N, Khvorova A. Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 30341012 DOI: 10.1016/J.Ymthe.2018.09.015 |
0.714 |
|
| 2018 |
Didiot MC, Ferguson CM, Ly S, Coles AH, Smith AO, Bicknell AA, Hall LM, Sapp E, Echeverria D, Pai AA, DiFiglia M, Moore MJ, Hayward LJ, Aronin N, Khvorova A. Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin. Cell Reports. 24: 2553-2560.e5. PMID 30184490 DOI: 10.1016/J.Celrep.2018.07.106 |
0.392 |
|
| 2018 |
Shmushkovich T, Monopoli KR, Homsy D, Leyfer D, Betancur-Boissel M, Khvorova A, Wolfson AD. Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs. Nucleic Acids Research. PMID 30169779 DOI: 10.1093/Nar/Gky745 |
0.375 |
|
| 2018 |
Godinho BMDC, Henninger N, Bouley J, Alterman JF, Haraszti RA, Gilbert JW, Sapp E, Coles AH, Biscans A, Nikan M, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 30143435 DOI: 10.1016/J.Ymthe.2018.08.005 |
0.752 |
|
| 2018 |
Mir A, Alterman JF, Hassler MR, Debacker AJ, Hudgens E, Echeverria D, Brodsky MH, Khvorova A, Watts JK, Sontheimer EJ. Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing. Nature Communications. 9: 2641. PMID 29980686 DOI: 10.1038/S41467-018-05073-Z |
0.461 |
|
| 2018 |
Haraszti RA, Miller R, Didiot MC, Biscans A, Alterman JF, Hassler MR, Roux L, Echeverria D, Sapp E, DiFiglia M, Aronin N, Khvorova A. Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29937418 DOI: 10.1016/J.Ymthe.2018.05.024 |
0.762 |
|
| 2018 |
Sharma VK, Osborn MF, Hassler M, Echeverria D, Ly S, Ulashchik E, Martynenko-Makaev Y, Shmanai VV, Zatsepin TS, Khvorova A, Watts J. Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo. Bioconjugate Chemistry. PMID 29898368 DOI: 10.1021/Acs.Bioconjchem.8B00365 |
0.328 |
|
| 2018 |
Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V. Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. Journal of Drug Delivery Science and Technology. 43: 453-460. PMID 29805475 DOI: 10.1016/J.Jddst.2017.11.013 |
0.748 |
|
| 2018 |
Osborn MF, Khvorova A. Improving Small Interfering RNA Delivery In Vivo Through Lipid Conjugation. Nucleic Acid Therapeutics. PMID 29746209 DOI: 10.1089/Nat.2018.0725 |
0.502 |
|
| 2018 |
Biscans A, Haraszti RA, Echeverria D, Miller R, Didiot MC, Nikan M, Roux L, Aronin N, Khvorova A. Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 29699940 DOI: 10.1016/J.Ymthe.2018.03.019 |
0.771 |
|
| 2018 |
Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A. Efficient gene silencing in brain tumors with hydrophobically modified siRNAs. Molecular Cancer Therapeutics. PMID 29654062 DOI: 10.1158/1535-7163.Mct-17-1144 |
0.392 |
|
| 2018 |
Hassler MR, Turanov AA, Alterman JF, Haraszti RA, Coles AH, Osborn MF, Echeverria D, Nikan M, Salomon WE, Roux L, Godinho BMDC, Davis SM, Morrissey DV, Zamore PD, Karumanchi SA, ... ... Khvorova A, et al. Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo. Nucleic Acids Research. PMID 29432571 DOI: 10.1093/Nar/Gky037 |
0.778 |
|
| 2018 |
Didiot MC, Haraszti RA, Aronin N, Khvorova A. Loading of Extracellular Vesicles with Hydrophobically Modified siRNAs. Methods in Molecular Biology (Clifton, N.J.). 1740: 199-214. PMID 29388146 DOI: 10.1007/978-1-4939-7652-2_16 |
0.759 |
|
| 2017 |
Godinho BMDC, Gilbert JW, Haraszti RA, Coles AH, Biscans A, Roux L, Nikan M, Echeverria D, Hassler M, Khvorova A. Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay. Nucleic Acid Therapeutics. PMID 29022758 DOI: 10.1089/Nat.2017.0690 |
0.761 |
|
| 2017 |
Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC. A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics. Bio-Protocol. 7. PMID 28966945 DOI: 10.21769/BioProtoc.2501 |
0.322 |
|
| 2017 |
Haraszti RA, Coles A, Aronin N, Khvorova A, Didiot MC. Loading of Extracellular Vesicles with Chemically Stabilized Hydrophobic siRNAs for the Treatment of Disease in the Central Nervous System. Bio-Protocol. 7. PMID 28868334 DOI: 10.21769/BioProtoc.2338 |
0.323 |
|
| 2017 |
Sharma VK, Singh SK, Krishnamurthy PM, Alterman JF, Haraszti RA, Khvorova A, Prasad AK, Watts JK. Synthesis and biological properties of triazole-linked locked nucleic acid. Chemical Communications (Cambridge, England). PMID 28736781 DOI: 10.1039/C7Cc04092J |
0.718 |
|
| 2017 |
Haraszti RA, Roux L, Coles AH, Turanov AA, Alterman JF, Echeverria D, Godinho BMDC, Aronin N, Khvorova A. 5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo. Nucleic Acids Research. PMID 28591791 DOI: 10.1093/Nar/Gkx507 |
0.767 |
|
| 2017 |
Nikan M, Osborn MF, Coles AH, Biscans A, Godinho B, Haraszti R, Sapp E, Echeverria D, DiFiglia M, Aronin N, Khvorova A. Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable, O-phosphocholine-N-docosahexaenoyl-L-serine siRNA Conjugate in Mouse Brain. Bioconjugate Chemistry. PMID 28462988 DOI: 10.1021/Acs.Bioconjchem.7B00226 |
0.789 |
|
| 2017 |
Khvorova A, Watts JK. The chemical evolution of oligonucleotide therapies of clinical utility. Nature Biotechnology. PMID 28244990 DOI: 10.1038/Nbt.3765 |
0.37 |
|
| 2017 |
Khvorova A. Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs. The New England Journal of Medicine. 376: 4-7. PMID 28052224 DOI: 10.1056/Nejmp1614154 |
0.4 |
|
| 2016 |
Ly S, Navaroli DM, Didiot MC, Cardia J, Pandarinathan L, Alterman JF, Fogarty K, Standley C, Lifshitz LM, Bellve KD, Prot M, Echeverria D, Corvera S, Khvorova A. Visualization of self-delivering hydrophobically modified siRNA cellular internalization. Nucleic Acids Research. PMID 27899655 DOI: 10.1093/Nar/Gkw1005 |
0.381 |
|
| 2016 |
Haraszti RA, Didiot MC, Sapp E, Leszyk J, Shaffer SA, Rockwell HE, Gao F, Narain NR, DiFiglia M, Kiebish MA, Aronin N, Khvorova A. High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources. Journal of Extracellular Vesicles. 5: 32570. PMID 27863537 DOI: 10.3402/Jev.V5.32570 |
0.722 |
|
| 2016 |
Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, ... Khvorova A, et al. Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 27506293 DOI: 10.1038/Mt.2016.126 |
0.798 |
|
| 2016 |
Nikan M, Osborn MF, Coles AH, Godinho BM, Hall LM, Haraszti RA, Hassler MR, Echeverria D, Aronin N, Khvorova A. Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain. Molecular Therapy. Nucleic Acids. 5: e344. PMID 27504598 DOI: 10.1038/Mtna.2016.50 |
0.789 |
|
| 2016 |
Coles AH, Osborn MF, Golebowski D, Hassler M, Esteves MS, Khvorova A. Abstract PR12: Robust modulation of gene expression in aggressive glioblastoma mouse models: A new approach for in vivo target validation Cancer Research. 76. DOI: 10.1158/1538-7445.Nonrna15-Pr12 |
0.429 |
|
| 2016 |
Khvorova A. Abstract IA27: Advances in oligonucleotide chemistry for the treatment of neurodegenerative disorders and brain tumors Cancer Research. 76. DOI: 10.1158/1538-7445.Nonrna15-Ia27 |
0.452 |
|
| 2015 |
Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Molecular Therapy. Nucleic Acids. 4: e266. PMID 26623938 DOI: 10.1038/Mtna.2015.38 |
0.414 |
|
| 2015 |
Coles AH, Osborn MF, Alterman JF, Turanov AA, Godinho BM, Kennington L, Chase K, Aronin N, Khvorova A. A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo. Nucleic Acid Therapeutics. PMID 26595721 DOI: 10.1089/Nat.2015.0578 |
0.757 |
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| 2015 |
Osborn MF, Alterman JF, Nikan M, Cao H, Didiot MC, Hassler MR, Coles AH, Khvorova A. Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs. Nucleic Acids Research. PMID 26400165 DOI: 10.1093/Nar/Gkv942 |
0.411 |
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| 2013 |
Byrne M, Tzekov R, Wang Y, Rodgers A, Cardia J, Ford G, Holton K, Pandarinathan L, Lapierre J, Stanney W, Bulock K, Shaw S, Libertine L, Fettes K, Khvorova A, et al. Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye. Journal of Ocular Pharmacology and Therapeutics : the Official Journal of the Association For Ocular Pharmacology and Therapeutics. 29: 855-64. PMID 24180627 DOI: 10.1089/Jop.2013.0148 |
0.403 |
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| 2012 |
Khvorova A, Wolfson A. New competition in RNA regulation. Nature Biotechnology. 30: 58-9. PMID 22231098 DOI: 10.1038/Nbt.2092 |
0.394 |
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| 2011 |
Lapierre J, Salomon W, Cardia J, Bulock K, Lam JT, Stanney WJ, Ford G, Smith-Anzures B, Woolf T, Kamens J, Khvorova A, Samarsky D. Potent and systematic RNAi mediated silencing with single oligonucleotide compounds. Rna (New York, N.Y.). 17: 1032-7. PMID 21493786 DOI: 10.1261/Rna.2399411 |
0.451 |
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| 2010 |
Robertson B, Dalby AB, Karpilow J, Khvorova A, Leake D, Vermeulen A. Specificity and functionality of microRNA inhibitors. Silence. 1: 10. PMID 20359337 DOI: 10.1186/1758-907X-1-10 |
0.354 |
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| 2008 |
Anderson E, Boese Q, Khvorova A, Karpilow J. Identifying siRNA-induced off-targets by microarray analysis. Methods in Molecular Biology (Clifton, N.J.). 442: 45-63. PMID 18369777 DOI: 10.1007/978-1-59745-191-8_4 |
0.406 |
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| 2008 |
Anderson EM, Birmingham A, Baskerville S, Reynolds A, Maksimova E, Leake D, Fedorov Y, Karpilow J, Khvorova A. Experimental validation of the importance of seed complement frequency to siRNA specificity. Rna (New York, N.Y.). 14: 853-61. PMID 18367722 DOI: 10.1261/Rna.704708 |
0.377 |
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| 2007 |
Birmingham A, Anderson E, Sullivan K, Reynolds A, Boese Q, Leake D, Karpilow J, Khvorova A. A protocol for designing siRNAs with high functionality and specificity. Nature Protocols. 2: 2068-78. PMID 17853862 DOI: 10.1038/Nprot.2007.278 |
0.412 |
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| 2007 |
Li L, Lin X, Khvorova A, Fesik SW, Shen Y. Defining the optimal parameters for hairpin-based knockdown constructs. Rna (New York, N.Y.). 13: 1765-74. PMID 17698642 DOI: 10.1261/Rna.599107 |
0.361 |
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| 2007 |
Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, Baskerville S, Maksimova E, Robinson K, Karpilow J, Marshall WS, Khvorova A. Correction: Corrigendum: 3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets Nature Methods. 4: 533-533. DOI: 10.1038/Nmeth0607-533 |
0.307 |
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| 2006 |
Jackson AL, Burchard J, Leake D, Reynolds A, Schelter J, Guo J, Johnson JM, Lim L, Karpilow J, Nichols K, Marshall W, Khvorova A, Linsley PS. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. Rna (New York, N.Y.). 12: 1197-205. PMID 16682562 DOI: 10.1261/Rna.30706 |
0.388 |
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| 2006 |
Fedorov Y, Anderson EM, Birmingham A, Reynolds A, Karpilow J, Robinson K, Leake D, Marshall WS, Khvorova A. Off-target effects by siRNA can induce toxic phenotype. Rna (New York, N.Y.). 12: 1188-96. PMID 16682561 DOI: 10.1261/Rna.28106 |
0.438 |
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| 2006 |
Reynolds A, Anderson EM, Vermeulen A, Fedorov Y, Robinson K, Leake D, Karpilow J, Marshall WS, Khvorova A. Induction of the interferon response by siRNA is cell type- and duplex length-dependent. Rna (New York, N.Y.). 12: 988-93. PMID 16611941 DOI: 10.1261/Rna.2340906 |
0.312 |
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| 2006 |
Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y, Baskerville S, Maksimova E, Robinson K, Karpilow J, Marshall WS, Khvorova A. 3' UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nature Methods. 3: 199-204. PMID 16489337 DOI: 10.1038/Nmeth854 |
0.42 |
|
| 2005 |
Benimetskaya L, Lai JC, Khvorova A, Wu S, Miller P, Stein CA. Induction of apoptosis by G3139 in melanoma cells. Annals of the New York Academy of Sciences. 1058: 235-45. PMID 16394140 DOI: 10.1196/Annals.1359.033 |
0.319 |
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| 2005 |
Vermeulen A, Behlen L, Reynolds A, Wolfson A, Marshall WS, Karpilow J, Khvorova A. The contributions of dsRNA structure to Dicer specificity and efficiency. Rna (New York, N.Y.). 11: 674-82. PMID 15811921 DOI: 10.1261/Rna.7272305 |
0.405 |
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| 2005 |
Fedorov Y, King A, Anderson E, Karpilow J, Ilsley D, Marshall W, Khvorova A. Different delivery methods-different expression profiles. Nature Methods. 2: 241. PMID 15782213 DOI: 10.1038/Nmeth0405-241 |
0.418 |
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| 2005 |
Boese Q, Leake D, Reynolds A, Read S, Scaringe SA, Marshall WS, Khvorova A. Mechanistic insights aid computational short interfering RNA design. Methods in Enzymology. 392: 73-96. PMID 15644176 DOI: 10.1016/S0076-6879(04)92005-8 |
0.347 |
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| 2004 |
Saksmerprome V, Roychowdhury-Saha M, Jayasena S, Khvorova A, Burke DH. Artificial tertiary motifs stabilize trans-cleaving hammerhead ribozymes under conditions of submillimolar divalent ions and high temperatures. Rna (New York, N.Y.). 10: 1916-24. PMID 15547137 DOI: 10.1261/Rna.7159504 |
0.331 |
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| 2004 |
Canny MD, Jucker FM, Kellogg E, Khvorova A, Jayasena SD, Pardi A. Fast cleavage kinetics of a natural hammerhead ribozyme. Journal of the American Chemical Society. 126: 10848-9. PMID 15339162 DOI: 10.1021/Ja046848V |
0.344 |
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| 2004 |
Raffo A, Lai JC, Stein CA, Miller P, Scaringe S, Khvorova A, Benimetskaya L. Antisense RNA down-regulation of bcl-2 expression in DU145 prostate cancer cells does not diminish the cytostatic effects of G3139 (Oblimersen). Clinical Cancer Research : An Official Journal of the American Association For Cancer Research. 10: 3195-206. PMID 15131061 DOI: 10.1158/1078-0432.Ccr-03-0287 |
0.374 |
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| 2004 |
Huang F, Khvorova A, Marshall W, Sorkin A. Analysis of clathrin-mediated endocytosis of epidermal growth factor receptor by RNA interference. The Journal of Biological Chemistry. 279: 16657-61. PMID 14985334 DOI: 10.1074/Jbc.C400046200 |
0.369 |
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| 2004 |
Hsieh AC, Bo R, Manola J, Vazquez F, Bare O, Khvorova A, Scaringe S, Sellers WR. A library of siRNA duplexes targeting the phosphoinositide 3-kinase pathway: determinants of gene silencing for use in cell-based screens. Nucleic Acids Research. 32: 893-901. PMID 14769947 DOI: 10.1093/Nar/Gkh238 |
0.406 |
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| 2004 |
Li T, Chang CY, Jin DY, Lin PJ, Khvorova A, Stafford DW. Identification of the gene for vitamin K epoxide reductase. Nature. 427: 541-4. PMID 14765195 DOI: 10.1038/Nature02254 |
0.343 |
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| 2004 |
Reynolds A, Leake D, Boese Q, Scaringe S, Marshall WS, Khvorova A. Rational siRNA design for RNA interference. Nature Biotechnology. 22: 326-30. PMID 14758366 DOI: 10.1038/Nbt936 |
0.463 |
|
| 2003 |
Khvorova A, Reynolds A, Jayasena SD. Functional siRNAs and miRNAs exhibit strand bias. Cell. 115: 209-16. PMID 14567918 DOI: 10.1016/S0092-8674(03)00801-8 |
0.348 |
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| 2003 |
Khvorova A, Lescoute A, Westhof E, Jayasena SD. Sequence elements outside the hammerhead ribozyme catalytic core enable intracellular activity. Nature Structural Biology. 10: 708-12. PMID 12881719 DOI: 10.1038/Nsb959 |
0.323 |
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| 2003 |
Wilson JA, Jayasena S, Khvorova A, Sabatinos S, Rodrigue-Gervais IG, Arya S, Sarangi F, Harris-Brandts M, Beaulieu S, Richardson CD. RNA interference blocks gene expression and RNA synthesis from hepatitis C replicons propagated in human liver cells. Proceedings of the National Academy of Sciences of the United States of America. 100: 2783-8. PMID 12594341 DOI: 10.1073/Pnas.252758799 |
0.394 |
|
| 2001 |
Vlassov A, Khvorova A, Yarus M. Binding and disruption of phospholipid bilayers by supramolecular RNA complexes Proceedings of the National Academy of Sciences of the United States of America. 98: 7706-7711. PMID 11427715 DOI: 10.1073/Pnas.141041098 |
0.383 |
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| 2001 |
Amaya E, Khvorova A, Piggot PJ. Analysis of promoter recognition in vivo directed by σF of Bacillus subtilis by using random-sequence oligonucleotides Journal of Bacteriology. 183: 3623-3630. PMID 11371526 DOI: 10.1128/Jb.183.12.3623-3630.2001 |
0.341 |
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| 1999 |
Khvorova A, Kwak YG, Tamkun M, Majerfeld I, Yarus M. RNAs that bind and change the permeability of phospholipid membranes Proceedings of the National Academy of Sciences of the United States of America. 96: 10649-10654. PMID 10485880 DOI: 10.1073/Pnas.96.19.10649 |
0.391 |
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