Antisense Drugs Make Sense for Neurological Diseases
Bennett, C. Frank, Kordasiewicz, Holly B, Cleveland, Don W
Published in Annual review of pharmacology and toxicology (06.01.2021)
Published in Annual review of pharmacology and toxicology (06.01.2021)
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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Kordasiewicz, Holly B., Stanek, Lisa M., Wancewicz, Edward V., Mazur, Curt, McAlonis, Melissa M., Pytel, Kimberly A., Artates, Jonathan W., Weiss, Andreas, Cheng, Seng H., Shihabuddin, Lamya S., Hung, Gene, Bennett, C. Frank, Cleveland, Don W.
Published in Neuron (Cambridge, Mass.) (21.06.2012)
Published in Neuron (Cambridge, Mass.) (21.06.2012)
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Targeting Huntingtin Expression in Patients with Huntington’s Disease
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
Published in The New England journal of medicine (13.06.2019)
Published in The New England journal of medicine (13.06.2019)
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Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice
McLoughlin, Hayley S., Moore, Lauren R., Chopra, Ravi, Komlo, Robert, McKenzie, Megan, Blumenstein, Kate G., Zhao, Hien, Kordasiewicz, Holly B., Shakkottai, Vikram G., Paulson, Henry L.
Published in Annals of neurology (01.07.2018)
Published in Annals of neurology (01.07.2018)
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Journal Article
A single-cell map of antisense oligonucleotide activity in the brain
Mortberg, Meredith A, Gentile, Juliana E, Nadaf, Naeem M, Vanderburg, Charles, Simmons, Sean, Dubinsky, Dan, Slamin, Adam, Maldonado, Salome, Petersen, Caroline L, Jones, Nichole, Kordasiewicz, Holly B, Zhao, Hien T, Vallabh, Sonia M, Minikel, Eric Vallabh
Published in Nucleic acids research (11.08.2023)
Published in Nucleic acids research (11.08.2023)
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Journal Article
Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy
DeVos, Sarah L, Miller, Rebecca L, Schoch, Kathleen M, Holmes, Brandon B, Kebodeaux, Carey S, Wegener, Amy J, Chen, Guo, Shen, Tao, Tran, Hien, Nichols, Brandon, Zanardi, Tom A, Kordasiewicz, Holly B, Swayze, Eric E, Bennett, C Frank, Diamond, Marc I, Miller, Timothy M
Published in Science translational medicine (25.01.2017)
Published in Science translational medicine (25.01.2017)
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Journal Article
Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints
Minikel, Eric Vallabh, Zhao, Hien T, Le, Jason, O’Moore, Jill, Pitstick, Rose, Graffam, Samantha, Carlson, George A, Kavanaugh, Michael P, Kriz, Jasna, Kim, Jae Beom, Ma, Jiyan, Wille, Holger, Aiken, Judd, McKenzie, Deborah, Doh-ura, Katsumi, Beck, Matthew, O’Keefe, Rhonda, Stathopoulos, Jacquelyn, Caron, Tyler, Schreiber, Stuart L, Carroll, Jeffrey B, Kordasiewicz, Holly B, Cabin, Deborah E, Vallabh, Sonia M
Published in Nucleic acids research (04.11.2020)
Published in Nucleic acids research (04.11.2020)
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Journal Article
ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43
Arnold, Eveline S, Ling, Shuo-Chien, Huelga, Stephanie C, Lagier-Tourenne, Clotilde, Polymenidou, Magdalini, Ditsworth, Dara, Kordasiewicz, Holly B, McAlonis-Downes, Melissa, Platoshyn, Oleksandr, Parone, Philippe A, Da Cruz, Sandrine, Clutario, Kevin M, Swing, Debbie, Tessarollo, Lino, Marsala, Martin, Shaw, Christopher E, Yeo, Gene W, Cleveland, Don W
Published in Proceedings of the National Academy of Sciences - PNAS (19.02.2013)
Published in Proceedings of the National Academy of Sciences - PNAS (19.02.2013)
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Journal Article
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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Journal Article
PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models
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
Published in The Journal of clinical investigation (02.01.2018)
Published in The Journal of clinical investigation (02.01.2018)
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Journal Article
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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Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43
Yeo, Gene W, Cleveland, Don W, Polymenidou, Magdalini, Lagier-Tourenne, Clotilde, Hutt, Kasey R, Huelga, Stephanie C, Moran, Jacqueline, Liang, Tiffany Y, Ling, Shuo-Chien, Sun, Eveline, Wancewicz, Edward, Mazur, Curt, Kordasiewicz, Holly, Sedaghat, Yalda, Donohue, John Paul, Shiue, Lily, Bennett, C Frank
Published in Nature neuroscience (01.04.2011)
Published in Nature neuroscience (01.04.2011)
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Journal Article
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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Journal Article
Neural deficits in a mouse model of PACS1 syndrome are corrected with PACS1- or HDAC6-targeting therapy
Villar-Pazos, Sabrina, Thomas, Laurel, Yang, Yunhan, Chen, Kun, Lyles, Jenea B., Deitch, Bradley J., Ochaba, Joseph, Ling, Karen, Powers, Berit, Gingras, Sebastien, Kordasiewicz, Holly B., Grubisha, Melanie J., Huang, Yanhua H., Thomas, Gary
Published in Nature communications (17.10.2023)
Published in Nature communications (17.10.2023)
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Journal Article
Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
Moore, Lauren R., Rajpal, Gautam, Dillingham, Ian T., Qutob, Maya, Blumenstein, Kate G., Gattis, Danielle, Hung, Gene, Kordasiewicz, Holly B., Paulson, Henry L., McLoughlin, Hayley S.
Published in Molecular therapy. Nucleic acids (16.06.2017)
Published in Molecular therapy. Nucleic acids (16.06.2017)
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Journal Article
In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides
Southwell, Amber L, Skotte, Niels H, Kordasiewicz, Holly B, Østergaard, Michael E, Watt, Andrew T, Carroll, Jeffrey B, Doty, Crystal N, Villanueva, Erika B, Petoukhov, Eugenia, Vaid, Kuljeet, Xie, Yuanyun, Freier, Susan M, Swayze, Eric E, Seth, Punit P, Bennett, Clarence Frank, Hayden, Michael R
Published in Molecular therapy (01.12.2014)
Published in Molecular therapy (01.12.2014)
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Journal Article
Alternative processing of human HTT mRNA with implications for Huntington's disease therapeutics
Fienko, Sandra, Landles, Christian, Sathasivam, Kirupa, McAteer, Sean J, Milton, Rebecca E, Osborne, Georgina F, Smith, Edward J, Jones, Samuel T, Bondulich, Marie K, Danby, Emily C E, Phillips, Jemima, Taxy, Bridget A, Kordasiewicz, Holly B, Bates, Gillian P
Published in Brain (London, England : 1878) (19.12.2022)
Published in Brain (London, England : 1878) (19.12.2022)
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Journal Article
Antisense oligonucleotides selectively suppress target RNA in nociceptive neurons of the pain system and can ameliorate mechanical pain
Mohan, Apoorva, Fitzsimmons, Bethany, Zhao, Hien T, Jiang, Yuhong, Mazur, Curt, Swayze, Eric E, Kordasiewicz, Holly B
Published in Pain (Amsterdam) (01.01.2018)
Published in Pain (Amsterdam) (01.01.2018)
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