Search Results - "Swayze, Eric"

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

“…Dramatic advances in understanding of the roles RNA plays in normal health and disease have greatly expanded over the past 10 years and have made it clear that…”
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Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression by 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.

“…Mutant huntingtin (HTT) protein causes Huntington disease (HD), an incurable neurological disorder. Silencing mutant HTT using nucleic acids would eliminate…”
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Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides by 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

“…Abstract The PS modification enhances the nuclease stability and protein binding properties of gapmer antisense oligonucleotides (ASOs) and is one of very few…”
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Targeting Huntingtin Expression in Patients with Huntington’s Disease by 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

“…Mutated HTT , resulting in mutant huntingtin, causes Huntington’s disease. A phase 1–2a trial of intrathecal delivery of an antisense oligonucleotide targeting…”
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Single-Stranded siRNAs Activate RNAi in Animals by 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.

“…The therapeutic utility of siRNAs is limited by the requirement for complex formulations to deliver them to tissues. If potent single-stranded RNAs could be…”
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Pharmacology of Antisense Drugs by Bennett, C. Frank, Baker, Brenda F, Pham, Nguyen, Swayze, Eric, Geary, Richard S

“…Recent studies have led to a greater appreciation of the diverse roles RNAs play in maintaining normal cellular function and how they contribute to disease…”
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Synthetic CRISPR RNA-Cas9–guided genome editing in human cells by Rahdar, Meghdad, McMahon, Moira A., Prakash, Thazha P., Swayze, Eric E., Bennett, C. Frank, Cleveland, Don W.

“…Genome editing with the clustered, regularly interspaced, short palindromic repeats (CRISPR)-Cas9 nuclease system is a powerful technology for manipulating…”
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Characterization of the interactions of chemically-modified therapeutic nucleic acids with plasma proteins using a fluorescence polarization assay by Gaus, Hans J, Gupta, Ruchi, Chappell, Alfred E, Østergaard, Michael E, Swayze, Eric E, Seth, Punit P

“…Abstract Interactions of chemically modified nucleic acid therapeutics with plasma proteins play an important role in facilitating distribution from the…”
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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice by 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

“…Triantennary N-acetyl galactosamine (GalNAc, GN3: ), a high-affinity ligand for the hepatocyte-specific asialoglycoprotein receptor (ASGPR), enhances the…”
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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 by 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

“…Abstract Antisense oligonucleotides (ASOs) have emerged as a new class of drugs to treat a wide range of diseases, including neurological indications…”
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Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle by 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

“…Abstract Enhancing the functional uptake of antisense oligonucleotide (ASO) in the muscle will be beneficial for developing ASO therapeutics targeting genes…”
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Site-specific incorporation of 5′-methyl DNA enhances the therapeutic profile of gapmer ASOs by 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

“…Abstract We recently showed that site-specific incorporation of 2′-modifications or neutral linkages in the oligo-deoxynucleotide gap region of toxic…”
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Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial by 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.

“…Tau plays a key role in Alzheimer’s disease (AD) pathophysiology, and accumulating evidence suggests that lowering tau may reduce this pathology. We sought to…”
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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides by 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

“…Therapeutic oligonucleotides are often modified using the phosphorothioate (PS) backbone modification which enhances stability from nuclease mediated…”
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LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model by 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.

“…No treatments exist to slow or halt Parkinson’s disease (PD) progression; however, inhibition of leucine-rich repeat kinase 2 (LRRK2) activity represents one…”
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Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice by Chappell, Alfred E, Gaus, Hans J, Berdeja, Andres, Gupta, Ruchi, Jo, Minji, Prakash, Thazha P, Oestergaard, Michael, Swayze, Eric E, Seth, Punit P

“…Conjugation of antisense oligonucleotide (ASO) with a variety of distinct lipophilic moieties like fatty acids and cholesterol increases ASO accumulation and…”
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PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models by Zhao, Hien Tran, Damle, Sagar, Ikeda-Lee, Karli, Kuntz, Steven, Li, Jian, Mohan, Apoorva, Kim, Aneeza, Hung, Gene, Scheideler, Mark A, Scherer, Steven S, Svaren, John, Swayze, Eric E, Kordasiewicz, Holly B

“…Charcot-Marie-Tooth disease type 1A (CMT1A) is caused by duplication of peripheral myelin protein 22 (PMP22) and is the most common hereditary peripheral…”
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Antisense oligonucleotides to therapeutically target SARS-CoV-2 infection by 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

“…Although the COVID-19 pandemic began over three years ago, the virus responsible for the disease, SARS-CoV-2, continues to infect people across the globe. As…”
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Systematic Investigation of Tether Length and Phosphorus Configuration in Backbone Constrained Macrocyclic Nucleic Acids to Modulate Binding Kinetics for RNA by 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

“…We recently described a chemical strategy to pre-organize a trinucleotide subunit in a conformation suitable for Watson–Crick base pairing for modulating the…”
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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients by 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

“…Huntington disease (HD) is an inherited, fatal neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. The mutant protein causes…”
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