A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system von Alterman, Julia F., Godinho, Bruno M. D. C., Hassler, Matthew R., Ferguson, Chantal M., Echeverria, Dimas, Sapp, Ellen, Haraszti, Reka A., Coles, Andrew H., Conroy, Faith, Miller, Rachael, Roux, Loic, Yan, Paul, Knox, Emily G., Turanov, Anton A., King, Robert M., Gernoux, Gwladys, Mueller, Christian, Gray-Edwards, Heather L., Moser, Richard P., Bishop, Nina C., Jaber, Samer M., Gounis, Matthew J., Sena-Esteves, Miguel, Pai, Athma A., DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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RNAi modulation of placental sFLT1 for the treatment of preeclampsia von Turanov, Anton A, Lo, Agnes, Hassler, Matthew R, Makris, Angela, Ashar-Patel, Ami, Alterman, Julia F, Coles, Andrew H, Haraszti, Reka A, Roux, Loic, Godinho, Bruno M D C, Echeverria, Dimas, Pears, Suzanne, Iliopoulos, Jim, Shanmugalingam, Renuka, Ogle, Robert, Zsengeller, Zsuzsanna K, Hennessy, Annemarie, Karumanchi, S Ananth, Moore, Melissa J, Khvorova, Anastasia

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Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways von Osborn, Maire F, Coles, Andrew H, Biscans, Annabelle, Haraszti, Reka A, Roux, Loic, Davis, Sarah, Ly, Socheata, Echeverria, Dimas, Hassler, Matthew R, Godinho, Bruno M D C, Nikan, Mehran, Khvorova, Anastasia

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Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles von Haraszti, Reka Agnes, Miller, Rachael, Didiot, Marie-Cecile, Biscans, Annabelle, Alterman, Julia F., Hassler, Matthew R., Roux, Loic, Echeverria, Dimas, Sapp, Ellen, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo von Hassler, Matthew R, Turanov, Anton A, Alterman, Julia F, Haraszti, Reka A, Coles, Andrew H, Osborn, Maire F, Echeverria, Dimas, Nikan, Mehran, Salomon, William E, Roux, Loïc, Godinho, Bruno M D C, Davis, Sarah M, Morrissey, David V, Zamore, Phillip D, Karumanchi, S Ananth, Moore, Melissa J, Aronin, Neil, Khvorova, Anastasia

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Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing von Mir, Aamir, Alterman, Julia F., Hassler, Matthew R., Debacker, Alexandre J., Hudgens, Edward, Echeverria, Dimas, Brodsky, Michael H., Khvorova, Anastasia, Watts, Jonathan K., Sontheimer, Erik J.

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Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing von Didiot, Marie-Cécile, Hall, Lauren M, Coles, Andrew H, Haraszti, Reka A, Godinho, Bruno MDC, Chase, Kathryn, Sapp, Ellen, Ly, Socheata, Alterman, Julia F, Hassler, Matthew R, Echeverria, Dimas, Raj, Lakshmi, Morrissey, David V, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models von Conroy, Faith, Miller, Rachael, Alterman, Julia F., Hassler, Matthew R., Echeverria, Dimas, Godinho, Bruno M. D. C., Knox, Emily G., Sapp, Ellen, Sousa, Jaquelyn, Yamada, Ken, Mahmood, Farah, Boudi, Adel, Kegel-Gleason, Kimberly, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Pfister, Edith L.

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An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis von Yenilmez, Batuhan, Wetoska, Nicole, Kelly, Mark, Echeverria, Dimas, Min, Kyounghee, Lifshitz, Lawrence, Alterman, Julia F., Hassler, Matthew R., Hildebrand, Samuel, DiMarzio, Chloe, McHugh, Nicholas, Vangjeli, Lorenc, Sousa, Jacquelyn, Pan, Meixia, Han, Xianlin, Brehm, Michael A., Khvorova, Anastasia, Czech, Michael P.

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Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo von Sharma, Vivek K, Osborn, Maire F, Hassler, Matthew R, Echeverria, Dimas, Ly, Socheata, Ulashchik, Egor A, Martynenko-Makaev, Yury V, Shmanai, Vadim V, Zatsepin, Timofei S, Khvorova, Anastasia, Watts, Jonathan K

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Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain von Alterman, Julia F, Hall, Lauren M, Coles, Andrew H, Hassler, Matthew R, Didiot, Marie-Cecile, Chase, Kathryn, Abraham, Jasmin, Sottosanti, Emily, Johnson, Emily, Sapp, Ellen, Osborn, Maire F, Difiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity von Yamada, Ken, Hildebrand, Samuel, Davis, Sarah M, Miller, Rachael, Conroy, Faith, Sapp, Ellen, Caiazzi, Jillian, Alterman, Julia F, Roux, Loic, Echeverria, Dimas, Hassler, Matthew R, Pfister, Edith L, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference von Ferguson, Chantal M., Echeverria, Dimas, Hassler, Matthew, Ly, Socheata, Khvorova, Anastasia

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Taking charge of siRNA delivery von Khvorova, Anastasia, Osborn, Maire F, Hassler, Matthew R

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Enhancing siRNA efficacy in vivo with extended nucleic acid backbones von Yamada, Ken, Hariharan, Vignesh N, Caiazzi, Jillian, Miller, Rachael, Ferguson, Chantal M, Sapp, Ellen, Fakih, Hassan H, Tang, Qi, Yamada, Nozomi, Furgal, Raymond C, Paquette, Joseph D, Biscans, Annabelle, Bramato, Brianna M, McHugh, Nicholas, Summers, Ashley, Lochmann, Clemens, Godinho, Bruno M D C, Hildebrand, Samuel, Jackson, Samuel O, Echeverria, Dimas, Hassler, Matthew R, Alterman, Julia F, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs von Osborn, Maire F, Alterman, Julia F, Nikan, Mehran, Cao, Hong, Didiot, Marie C, Hassler, Matthew R, Coles, Andrew H, Khvorova, Anastasia

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Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition von Haraszti, Reka Agnes, Miller, Rachael, Dubuke, Michelle L, Rockwell, Hannah E, Coles, Andrew H, Sapp, Ellen, Didiot, Marie-Cecile, Echeverria, Dimas, Stoppato, Matteo, Sere, Yves Y, Leszyk, John, Alterman, Julia F, Godinho, Bruno M D C, Hassler, Matthew R, McDaniel, Justice, Narain, Niven R, Wollacott, Rachel, Wang, Yang, Shaffer, Scott A, Kiebish, Michael A, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain von Nikan, Mehran, Osborn, Maire F, Coles, Andrew H, Godinho, Bruno MDC, Hall, Lauren M, Haraszti, Reka A, Hassler, Matthew R, Echeverria, Dimas, Aronin, Neil, Khvorova, Anastasia

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PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs von Godinho, Bruno M.D.C., Knox, Emily G., Hildebrand, Samuel, Gilbert, James W., Echeverria, Dimas, Kennedy, Zachary, Haraszti, Reka A., Ferguson, Chantal M., Coles, Andrew H., Biscans, Annabelle, Caiazzi, Jillian, Alterman, Julia F., Hassler, Matthew R., Khvorova, Anastasia

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2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA von Davis, Sarah M., Sousa, Jacquelyn, Vangjeli, Lorenc, Hassler, Matthew R., Echeverria, Dimas, Knox, Emily, Turanov, Anton A., Alterman, Julia F., Khvorova, Anastasia

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