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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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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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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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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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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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides von Østergaard, 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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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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Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor von Schmidt, Karsten, Prakash, Thazha P, Donner, Aaron J, Kinberger, Garth A, Gaus, Hans J, Low, Audrey, Østergaard, Michael E, Bell, Melanie, Swayze, Eric E, Seth, Punit P

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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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Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1 von Pandey, Sanjay K, Wheeler, Thurman M, Justice, Samantha L, Kim, Aneeza, Younis, Husam S, Gattis, Danielle, Jauvin, Dominic, Puymirat, Jack, Swayze, Eric E, Freier, Susan M, Bennett, C Frank, Thornton, Charles A, MacLeod, A Robert

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Systematic Investigation of Tether Length and Phosphorus Configuration in Backbone Constrained Macrocyclic Nucleic Acids to Modulate Binding Kinetics for RNA von Rajasekaran, Tamilselvan, Freestone, Graeme C., Galindo-Murillo, Rodrigo, Lugato, Barbara, Gaus, Hans, Migawa, Michael T., Swayze, Eric. E., Cheatham, Thomas E., Seth, Punit P., Hanessian, Stephen

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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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Backbone Hydrocarbon-Constrained Nucleic Acids Modulate Hybridization Kinetics for RNA von Rajasekaran, Tamilselvan, Freestone, Graeme C, Galindo-Murillo, Rodrigo, Lugato, Barbara, Rico, Lorena, Salinas, Juan C, Gaus, Hans, Migawa, Michael T, Swayze, Eric E, Cheatham, Thomas E, Hanessian, Stephen, Seth, Punit P

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Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages von Wan, W Brad, Migawa, Michael T, Vasquez, Guillermo, Murray, Heather M, Nichols, Josh G, Gaus, Hans, Berdeja, Andres, Lee, Sam, Hart, Christopher E, Lima, Walt F, Swayze, Eric E, Seth, Punit P

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