Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression
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.
Published in Cell (31.08.2012)
Published in Cell (31.08.2012)
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Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides
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
Published in Nucleic acids research (20.09.2021)
Published in Nucleic acids research (20.09.2021)
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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
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
Published in Nucleic acids research (29.07.2014)
Published in Nucleic acids research (29.07.2014)
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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
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
Published in Nucleic acids research (25.01.2021)
Published in Nucleic acids research (25.01.2021)
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Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle
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
Published in Nucleic acids research (09.07.2019)
Published in Nucleic acids research (09.07.2019)
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Single-Stranded siRNAs Activate RNAi in Animals
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.
Published in Cell (31.08.2012)
Published in Cell (31.08.2012)
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Site-specific incorporation of 5′-methyl DNA enhances the therapeutic profile of gapmer ASOs
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
Published in Nucleic acids research (26.02.2021)
Published in Nucleic acids research (26.02.2021)
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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides
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
Published in Nucleic acids research (28.02.2020)
Published in Nucleic acids research (28.02.2020)
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LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model
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.
Published in Molecular therapy. Nucleic acids (15.09.2017)
Published in Molecular therapy. Nucleic acids (15.09.2017)
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Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice
Chappell, Alfred E, Gaus, Hans J, Berdeja, Andres, Gupta, Ruchi, Jo, Minji, Prakash, Thazha P, Oestergaard, Michael, Swayze, Eric E, Seth, Punit P
Published in Nucleic acids research (07.05.2020)
Published in Nucleic acids research (07.05.2020)
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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
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
Published in Nucleic acids research (18.03.2016)
Published in Nucleic acids research (18.03.2016)
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Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor
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
Published in Nucleic acids research (17.03.2017)
Published in Nucleic acids research (17.03.2017)
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Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7
Niu, Chenchen, Prakash, Thazah P, Kim, Aneeza, Quach, John L, Huryn, Laryssa A, Yang, Yuechen, Lopez, Edith, Jazayeri, Ali, Hung, Gene, Sopher, Bryce L, Brooks, Brian P, Swayze, Eric E, Bennett, C Frank, La Spada, Albert R
Published in Science translational medicine (31.10.2018)
Published in Science translational medicine (31.10.2018)
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Tcf4 Regulates Synaptic Plasticity, DNA Methylation, and Memory Function
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
Published in Cell reports (Cambridge) (06.09.2016)
Published in Cell reports (Cambridge) (06.09.2016)
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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
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
Published in The Journal of pharmacology and experimental therapeutics (01.11.2015)
Published in The Journal of pharmacology and experimental therapeutics (01.11.2015)
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Systematic Investigation of Tether Length and Phosphorus Configuration in Backbone Constrained Macrocyclic Nucleic Acids to Modulate Binding Kinetics for RNA
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
Published in Journal of organic chemistry (17.03.2023)
Published in Journal of organic chemistry (17.03.2023)
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Journal Article
Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease
Southwell, Amber L, Kordasiewicz, Holly B, Langbehn, Douglas, Skotte, Niels H, Parsons, Matthew P, Villanueva, Erika B, Caron, Nicholas S, Østergaard, Michael E, Anderson, Lisa M, Xie, Yuanyun, Cengio, Louisa Dal, Findlay-Black, Hailey, Doty, Crystal N, Fitsimmons, Bethany, Swayze, Eric E, Seth, Punit P, Raymond, Lynn A, Frank Bennett, C, Hayden, Michael R
Published in Science translational medicine (03.10.2018)
Published in Science translational medicine (03.10.2018)
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Backbone Hydrocarbon-Constrained Nucleic Acids Modulate Hybridization Kinetics for RNA
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
Published in Journal of the American Chemical Society (02.02.2022)
Published in Journal of the American Chemical Society (02.02.2022)
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