A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons in vitro with Oligonucleotide Therapeutics

di Alterman, Julia F., Coles, Andrew H., Hall, Lauren M., Aronin, Neil, Khvorova, Anastasia, Didiot, Marie-Cécile
Pubblicato in Bio Protoc (2017)

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

di Osborn, Maire F., Alterman, Julia F., Nikan, Mehran, Cao, Hong, Didiot, Marie C., Hassler, Matthew R., Coles, Andrew H., Khvorova, Anastasia
Pubblicato in Nucleic Acids Res (2015)

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

di Davis, Sarah M., Sousa, Jacquelyn, Vangjeli, Lorenc, Hassler, Matthew R., Echeverria, Dimas, Knox, Emily, Turanov, Anton A., Alterman, Julia F., Khvorova, Anastasia
Pubblicato in Mol Ther Nucleic Acids (2020)

A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo

di Coles, Andrew H., Osborn, Maire F., Alterman, Julia F., Turanov, Anton A., Godinho, Bruno M.D.C., Kennington, Lori, Chase, Kathryn, Aronin, Neil, Khvorova, Anastasia
Pubblicato in Nucleic Acid Ther (2016)

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

di 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
Pubblicato in Nucleic Acids Res (2017)

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

di 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.
Pubblicato in Nat Commun (2018)

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

di 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
Pubblicato in Nucleic Acids Res (2017)

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

di 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
Pubblicato in Mol Ther Nucleic Acids (2015)

Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier

di 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
Pubblicato in Mol Ther (2018)

Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing

di 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
Pubblicato in Mol Ther (2016)

RNAi Modulation of Placental sFLT1 for the Treatment of Preeclampsia.

di 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
Pubblicato in Nat Biotechnol (2018)

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

di 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
Pubblicato in Nucleic Acids Res (2018)

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

di 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
Pubblicato in iScience (2019)

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

di 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
Pubblicato in Nat Biotechnol (2019)