RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform von BENNETT, C. Frank, SWAYZE, Eric E

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Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression von Yu, Dongbo, Pendergraff, Hannah, Liu, Jing, Kordasiewicz, Holly B., Cleveland, Don W., Swayze, Eric E., Lima, Walt F., Crooke, Stanley T., Prakash, Thazha P., Corey, David R.

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Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides von Anderson, Brooke A, Freestone, Graeme C, Low, Audrey, De-Hoyos, Cheryl L, III, William J Drury, Østergaard, Michael E, Migawa, Michael T, Fazio, Michael, Wan, W Brad, Berdeja, Andres, Scandalis, Eli, Burel, Sebastien A, Vickers, Timothy A, Crooke, Stanley T, Swayze, Eric E, Liang, Xuehai, Seth, Punit P

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Single-Stranded siRNAs Activate RNAi in Animals von Lima, Walt F., Prakash, Thazha P., Murray, Heather M., Kinberger, Garth A., Li, Wenyu, Chappell, Alfred E., Li, Cheryl S., Murray, Susan F., Gaus, Hans, Seth, Punit P., Swayze, Eric E., Crooke, Stanley T.

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Synthetic CRISPR RNA-Cas9–guided genome editing in human cells von Rahdar, Meghdad, McMahon, Moira A., Prakash, Thazha P., Swayze, Eric E., Bennett, C. Frank, Cleveland, Don W.

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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice von Prakash, Thazha P, Graham, Mark J, Yu, Jinghua, Carty, Rick, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Zhao, Chenguang, Aghajan, Mariam, Murray, Heather F, Riney, Stan, Booten, Sheri L, Murray, Susan F, Gaus, Hans, Crosby, Jeff, Lima, Walt F, Guo, Shuling, Monia, Brett P, Swayze, Eric E, Seth, Punit P

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Targeting Huntingtin Expression in Patients with Huntington’s Disease von Tabrizi, Sarah J, Leavitt, Blair R, Landwehrmeyer, G. Bernhard, Wild, Edward J, Saft, Carsten, Barker, Roger A, Blair, Nick F, Craufurd, David, Priller, Josef, Rickards, Hugh, Rosser, Anne, Kordasiewicz, Holly B, Czech, Christian, Swayze, Eric E, Norris, Daniel A, Baumann, Tiffany, Gerlach, Irene, Schobel, Scott A, Paz, Erika, Smith, Anne V, Bennett, C. Frank, Lane, Roger M

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The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration von Jafar-nejad, Paymaan, Powers, Berit, Soriano, Armand, Zhao, Hien, Norris, Daniel A, Matson, John, DeBrosse-Serra, Beatrice, Watson, Jamie, Narayanan, Padmakumar, Chun, Seung J, Mazur, Curt, Kordasiewicz, Holly, Swayze, Eric E, Rigo, Frank

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Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle von Prakash, Thazha P, Mullick, Adam E, Lee, Richard G, Yu, Jinghua, Yeh, Steve T, Low, Audrey, Chappell, Alfred E, Østergaard, Michael E, Murray, Sue, Gaus, Hans J, Swayze, Eric E, Seth, Punit P

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Pharmacology of Antisense Drugs von Bennett, C. Frank, Baker, Brenda F, Pham, Nguyen, Swayze, Eric, Geary, Richard S

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Site-specific incorporation of 5′-methyl DNA enhances the therapeutic profile of gapmer ASOs von Vasquez, Guillermo, Freestone, Graeme C, Wan, W Brad, Low, Audrey, De Hoyos, Cheryl Li, Yu, Jinghua, Prakash, Thazha P, Ǿstergaard, Michael E, Liang, Xue-hai, Crooke, Stanley T, Swayze, Eric E, Migawa, Michael T, Seth, Punit P

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Characterization of the interactions of chemically-modified therapeutic nucleic acids with plasma proteins using a fluorescence polarization assay von Gaus, Hans J, Gupta, Ruchi, Chappell, Alfred E, Østergaard, Michael E, Swayze, Eric E, Seth, Punit P

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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides von Ostergaard, Michael E., De Hoyos, Cheryl L., Wan, W. Brad, Shen, Wen, Low, Audrey, Berdeja, Andres, Vasquez, Guillermo, Murray, Susan, Migawa, Michael T., Liang, Xue-hai, Swayze, Eric E., Crooke, Stanley T., Seth, Punit P.

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LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model von Zhao, Hien Tran, John, Neena, Delic, Vedad, Ikeda-Lee, Karli, Kim, Aneeza, Weihofen, Andreas, Swayze, Eric E., Kordasiewicz, Holly B., West, Andrew B., Volpicelli-Daley, Laura A.

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Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice von Chappell, Alfred E., Gaus, Hans J., Berdeja, Andres, Gupta, Ruchi, Jo, Minji, Prakash, Thazha P., Oestergaard, Michael, Swayze, Eric E., Seth, Punit P.

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Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial von Mummery, Catherine J., Börjesson-Hanson, Anne, Blackburn, Daniel J., Vijverberg, Everard G. B., De Deyn, Peter Paul, Ducharme, Simon, Jonsson, Michael, Schneider, Anja, Rinne, Juha O., Ludolph, Albert C., Bodenschatz, Ralf, Kordasiewicz, Holly, Swayze, Eric E., Fitzsimmons, Bethany, Mignon, Laurence, Moore, Katrina M., Yun, Chris, Baumann, Tiffany, Li, Dan, Norris, Daniel A., Crean, Rebecca, Graham, Danielle L., Huang, Ellen, Ratti, Elena, Bennett, C. Frank, Junge, Candice, Lane, Roger M.

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Antisense oligonucleotides to therapeutically target SARS-CoV-2 infection von Qiao, Yuanyuan, Wotring, Jesse W, Zhang, Charles J, Jiang, Xia, Xiao, Lanbo, Watt, Andy, Gattis, Danielle, Scandalis, Eli, Freier, Susan, Zheng, Yang, Pretto, Carla D, Ellison, Stephanie J, Swayze, Eric E, Guo, Shuling, Sexton, Jonathan Z, Chinnaiyan, Arul M

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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients von Skotte, Niels H, Southwell, Amber L, Østergaard, Michael E, Carroll, Jeffrey B, Warby, Simon C, Doty, Crystal N, Petoukhov, Eugenia, Vaid, Kuljeet, Kordasiewicz, Holly, Watt, Andrew T, Freier, Susan M, Hung, Gene, Seth, Punit P, Bennett, C Frank, Swayze, Eric E, Hayden, Michael R

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Tcf4 Regulates Synaptic Plasticity, DNA Methylation, and Memory Function von Kennedy, Andrew J., Rahn, Elizabeth J., Paulukaitis, Brynna S., Savell, Katherine E., Kordasiewicz, Holly B., Wang, Jing, Lewis, John W., Posey, Jessica, Strange, Sarah K., Guzman-Karlsson, Mikael C., Phillips, Scott E., Decker, Kyle, Motley, S. Timothy, Swayze, Eric E., Ecker, David J., Michael, Todd P., Day, Jeremy J., Sweatt, J. David

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Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts von Burel, Sebastien A, Hart, Christopher E, Cauntay, Patrick, Hsiao, Jill, Machemer, Todd, Katz, Melanie, Watt, Andy, Bui, Huynh-Hoa, Younis, Husam, Sabripour, Mahyar, Freier, Susan M, Hung, Gene, Dan, Amy, Prakash, T P, Seth, Punit P, Swayze, Eric E, Bennett, C Frank, Crooke, Stanley T, Henry, Scott P

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