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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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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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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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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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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Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin von Tang, Qi, Fakih, Hassan H., Zain UI Abideen, Mohammad, Hildebrand, Samuel R., Afshari, Khashayar, Gross, Katherine Y., Sousa, Jacquelyn, Maebius, Allison S., Bartholdy, Christina, Søgaard, Pia Pernille, Jackerott, Malene, Hariharan, Vignesh, Summers, Ashley, Fan, Xueli, Okamura, Ken, Monopoli, Kathryn R., Cooper, David A., Echeverria, Dimas, Bramato, Brianna, McHugh, Nicholas, Furgal, Raymond C., Dresser, Karen, Winter, Sarah J., Biscans, Annabelle, Chuprin, Jane, Haddadi, Nazgol-Sadat, Sherman, Shany, Yıldız-Altay, Ümmügülsüm, Rashighi, Mehdi, Richmond, Jillian M., Bouix-Peter, Claire, Blanchard, Carine, Clauss, Adam, Alterman, Julia F., Khvorova, Anastasia, Harris, John E.

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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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Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1 von Davis, Sarah M., Hariharan, Vignesh N., Lo, Agnes, Turanov, Anton A., Echeverria, Dimas, Sousa, Jacquelyn, McHugh, Nicholas, Biscans, Annabelle, Alterman, Julia F., Karumanchi, S. Ananth, Moore, Melissa J., Khvorova, Anastasia

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Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors von Cheng, Shun-Yun, Caiazzi, Jillian, Biscans, Annabelle, Alterman, Julia F., Echeverria, Dimas, McHugh, Nicholas, Hassler, Matthew, Jolly, Samson, Giguere, Delaney, Cipi, Joris, Khvorova, Anastasia, Punzo, Claudio

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Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier von Godinho, Bruno M.D.C., Henninger, Nils, Bouley, James, Alterman, Julia F., Haraszti, Reka A., Gilbert, James W., Sapp, Ellen, Coles, Andrew H., Biscans, Annabelle, Nikan, Mehran, Echeverria, Dimas, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia

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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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Multispecies-targeting siRNAs for the modulation of JAK1 in the skin von Tang, Qi, Gross, Katherine Y., Fakih, Hassan H., Jackson, Samuel O., Zain U.I. Abideen, Mohammad, Monopoli, Kathryn R., Blanchard, Carine, Bouix-Peter, Claire, Portal, Thibaud, Harris, John E., Khvorova, Anastasia, Alterman, Julia F.

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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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Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease von McAdam, Robyn L., Morton, Andrew, Gordon, Sarah L., Alterman, Julia F., Khvorova, Anastasia, Cousin, Michael A., Smillie, Karen J.

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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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Visualization of self-delivering hydrophobically modified siRNA cellular internalization von Ly, Socheata, Navaroli, Deanna M., Didiot, Marie-Cécile, Cardia, James, Pandarinathan, Lakshmipathi, Alterman, Julia F., Fogarty, Kevin, Standley, Clive, Lifshitz, Lawrence M., Bellve, Karl D., Prot, Matthieu, Echeverria, Dimas, Corvera, Silvia, Khvorova, Anastasia

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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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5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo von Haraszti, Reka A, Roux, Loic, Coles, Andrew H, Turanov, Anton A, Alterman, Julia F, Echeverria, Dimas, Godinho, Bruno M D C, Aronin, Neil, Khvorova, Anastasia

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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep von Ferguson, Chantal M, Godinho, Bruno Mdc, Alterman, Julia F, Coles, Andrew H, Hassler, Matthew, Echeverria, Dimas, Gilbert, James W, Knox, Emily G, Caiazzi, Jillian, Haraszti, Reka A, King, Robert M, Taghian, Toloo, Puri, Ajit, Moser, Richard P, Gounis, Matthew J, Aronin, Neil, Gray-Edwards, Heather, Khvorova, Anastasia

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