DIPG-17. TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA USING ALLELE-SPECIFIC SIRNA TARGETING H3.3 K27M MUTANT HISTONE

por Wentworth, Simon, Batista, Ana Rita, Alterman, Julia, Khvorova, Anastasia, Sena-Esteves, Miguel
Publicado 2023

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs

por Osborn, Maire F., Alterman, Julia F., Nikan, Mehran, Cao, Hong, Didiot, Marie C., Hassler, Matthew R., Coles, Andrew H., Khvorova, Anastasia
Publicado 2015

2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA

por Davis, Sarah M., Sousa, Jacquelyn, Vangjeli, Lorenc, Hassler, Matthew R., Echeverria, Dimas, Knox, Emily, Turanov, Anton A., Alterman, Julia F., Khvorova, Anastasia
Publicado 2020

5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

por 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
Publicado 2017

Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing

por 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.
Publicado 2018

Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1

por 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
Publicado 2022

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

por 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
Publicado 2017

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

por 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
Publicado 2015

Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity

por 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
Publicado 2021

PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs

por 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
Publicado 2022

An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis

por 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.
Publicado 2022

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

por 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.
Publicado 2022

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo

por Hariharan, Vignesh Narayan, Caiazzi, Jillian, Miller, Rachael, Ferguson, Chantal, Sapp, Ellen, Fakih, Hassan, Tang, Qi, Yamada, Nozomi, Furgal, Raymond, Paquette, Joseph, Bramato, Brianna, McHugh, Nicholas, Summers, Ashley, Lochmann, Clemens, Godinho, Bruno, Hildebrand, Samuel, Echeverria, Dimas, Hassler, Matthew, Alterman, Julia, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia, Yamada, Ken
Publicado 2023

RNAi Modulation of Placental sFLT1 for the Treatment of Preeclampsia.

por 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 MDC, Echeverria, Dimas, Pears, Suzanne, Iliopoulos, Jim, Shanmugalingam, Renuka, Ogle, Robert, Zsengeller, Zsuzsanna K., Hennessy, Annemarie, Karumanchi, S. Ananth, Moore, Melissa J., Khvorova, Anastasia
Publicado 2018

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

por Ferguson, Chantal M., Godinho, Bruno M.D.C., 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
Publicado 2021

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

por 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
Publicado 2018

Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.

por Yamada, Ken, Hariharan, Vignesh Narayan, Caiazzi, Jillian, Miller, Rachael, Furguson, Chantal, Sapp, Ellen, Fakih, Hassan, Tan, Qi, Yamada, Nozomi, Furgal, Raymond C., Paquette, Joseph, Bramato, Brianna, McHugh, Nicholas, Summers, Ashley, Lochmann, Clemens, Godinho, Bruno M.D.C., Hildebrand, Samuel, Echeverria, Dimas, Hassler, Matthew R., Alterman, Julia F., DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Publicado 2023

Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition

por 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
Publicado 2019

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

por 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
Publicado 2019

Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin

por 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.
Publicado 2023