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

Lessons Learned from Open-label Deep Brain Stimulation for Tourette Syndrome: Eight Cases over 7 Years

por Motlagh, Maria G., Smith, Megan E., Landeros-Weisenberger, Angeli, Kobets, Andrew J., King, Robert A., Miravite, Joan, de Lotbinière, Alain C. J., Alterman, Ron L., Mogilner, Alon Y., Pourfar, Michael H., Okun, Michael S., Leckman, James F.
Publicado 2013

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

Tumor-Derived Cell Culture Model for the Investigation of Meningioma Biology

por Uhlmann, Erik J, Rabinovsky, Rosalia, Varma, Hemant, El Fatimy, Rachid, Kasper, Ekkehard M, Moore, Justin M, Vega, Rafael A, Thomas, Ajith J, Alterman, Ronald L, Stippler, Martina, Anderson, Matthew P, Uhlmann, Erik N, Kipper, Franciele C, Krichevsky, Anna M
Publicado 2021

Correction to: Tumor-Derived Cell Culture Model for the Investigation of Meningioma Biology

por Uhlmann, Erik J, Rabinovsky, Rosalia, Varma, Hemant, El Fatimy, Rachid, Kasper, Ekkehard M, Moore, Justin M, Vega, Rafael A, Thomas, Ajith J, Alterman, Ronald L, Stippler, Martina, Anderson, Matthew P, Uhlmann, Erik N, Kipper, Franciela C, Krichevsky, Anna M
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

Defining the Middle Corona

por West, Matthew J., Seaton, Daniel B., Wexler, David B., Raymond, John C., Del Zanna, Giulio, Rivera, Yeimy J., Kobelski, Adam R., Chen, Bin, DeForest, Craig, Golub, Leon, Caspi, Amir, Gilly, Chris R., Kooi, Jason E., Meyer, Karen A., Alterman, Benjamin L., Alzate, Nathalia, Andretta, Vincenzo, Auchère, Frédéric, Banerjee, Dipankar, Berghmans, David, Chamberlin, Phillip, Chitta, Lakshmi Pradeep, Downs, Cooper, Giordano, Silvio, Harra, Louise, Higginson, Aleida, Howard, Russell A., Kumar, Pankaj, Mason, Emily, Mason, James P., Morton, Richard J., Nykyri, Katariina, Patel, Ritesh, Rachmeler, Laurel, Reardon, Kevin P., Reeves, Katharine K., Savage, Sabrina, Thompson, Barbara J., Van Kooten, Samuel J., Viall, Nicholeen M., Vourlidas, Angelos, Zhukov, Andrei N.
Publicado 2023

Proceedings of the Second Annual Deep Brain Stimulation Think Tank: What's in the Pipeline

por Gunduz, Aysegul, Morita, Hokuto, Rossi, P. Justin, Allen, William L., Alterman, Ron L., Bronte-Stewart, Helen, Butson, Christopher R., Charles, David, Deckers, Sjaak, de Hemptinne, Coralie, DeLong, Mahlon, Dougherty, Darin, Ellrich, Jens, Foote, Kelly D., Giordano, James, Goodman, Wayne, Greenberg, Benjamin D., Greene, David, Gross, Robert, Judy, Jack W., Karst, Edward, Kent, Alexander, Kopell, Brian, Lang, Anthony, Lozano, Andres, Lungu, Codrin, Lyons, Kelly E., Machado, Andre, Martens, Hubert, McIntyre, Cameron, Min, Hoon-Ki, Neimat, Joseph, Ostrem, Jill, Pannu, Sat, Ponce, Francisco, Pouratian, Nader, Reymers, Donnie, Schrock, Lauren, Sheth, Sameer, Shih, Ludy, Stanslaski, Scott, Steinke, G. Karl, Stypulkowski, Paul, Tröster, Alexander I., Verhagen, Leo, Walker, Harrison, Okun, Michael S.
Publicado 2015

Proceedings of the Third Annual Deep Brain Stimulation Think Tank: A Review of Emerging Issues and Technologies

por Rossi, P. Justin, Gunduz, Aysegul, Judy, Jack, Wilson, Linda, Machado, Andre, Giordano, James J., Elias, W. Jeff, Rossi, Marvin A., Butson, Christopher L., Fox, Michael D., McIntyre, Cameron C., Pouratian, Nader, Swann, Nicole C., de Hemptinne, Coralie, Gross, Robert E., Chizeck, Howard J., Tagliati, Michele, Lozano, Andres M., Goodman, Wayne, Langevin, Jean-Philippe, Alterman, Ron L., Akbar, Umer, Gerhardt, Greg A., Grill, Warren M., Hallett, Mark, Herrington, Todd, Herron, Jeffrey, van Horne, Craig, Kopell, Brian H., Lang, Anthony E., Lungu, Codrin, Martinez-Ramirez, Daniel, Mogilner, Alon Y., Molina, Rene, Opri, Enrico, Otto, Kevin J., Oweiss, Karim G., Pathak, Yagna, Shukla, Aparna, Shute, Jonathan, Sheth, Sameer A., Shih, Ludy C., Steinke, G. Karl, Tröster, Alexander I., Vanegas, Nora, Zaghloul, Kareem A., Cendejas-Zaragoza, Leopoldo, Verhagen, Leonard, Foote, Kelly D., Okun, Michael S.
Publicado 2016