Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis von 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.

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Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice von 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.

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Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints von 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

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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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ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43 von 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

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A single-cell map of antisense oligonucleotide activity in the brain von 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

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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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Neural deficits in a mouse model of PACS1 syndrome are corrected with PACS1- or HDAC6-targeting therapy von 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

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In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides von 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

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Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models von 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.

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Antisense oligonucleotides selectively suppress target RNA in nociceptive neurons of the pain system and can ameliorate mechanical pain von Mohan, Apoorva, Fitzsimmons, Bethany, Zhao, Hien T., Jiang, Yuhong, Mazur, Curt, Swayze, Eric E., Kordasiewicz, Holly B.

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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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Antisense Drugs Make Sense for Neurological Diseases von Bennett, C. Frank, Kordasiewicz, Holly B, Cleveland, Don W

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alpha-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease von Cole, Tracy A., Zhao, Hien, Collier, Timothy J., Sandoval, Ivette, Sortwell, Caryl E., Steece-Collier, Kathy, Daley, Brian F., Booms, Alix, Lipton, Jack, Welch, Mackenzie, Berman, Melissa, Jandreski, Luke, Graham, Danielle, Weihofen, Andreas, Celano, Stephanie, Schulz, Emily, Cole-Strauss, Allyson, Luna, Esteban, Quach, Duc, Mohan, Apoorva, Bennett, C. Frank, Swayze, Eric E., Kordasiewicz, Holly B., Luk, Kelvin C., Paumier, Katrina L.

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Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment von O’Callaghan, Brennon, Hofstra, Bente, Handler, Hillary P., Kordasiewicz, Holly B., Cole, Tracy, Duvick, Lisa, Friedrich, Jillian, Rainwater, Orion, Yang, Praseuth, Benneyworth, Michael, Nichols-Meade, Tessa, Heal, Wesley, Ter Haar, Rachel, Henzler, Christine, Orr, Harry T.

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Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks von Leavitt, Blair R, Kordasiewicz, Holly B, Schobel, Scott A

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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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Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia von Keiser, Megan S, Kordasiewicz, Holly B, McBride, Jodi L

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Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.HttQ111/+ mouse model of Huntington's disease von Coffey, Sydney R, Bragg, Robert M, Minnig, Shawn, Ament, Seth A, Cantle, Jeffrey P, Glickenhaus, Anne, Shelnut, Daniel, Carrillo, José M, Shuttleworth, Dominic D, Rodier, Julie-Anne, Noguchi, Kimihiro, Bennett, C Frank, Price, Nathan D, Kordasiewicz, Holly B, Carroll, Jeffrey B

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Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNS von Mortberg, Meredith A, Zhao, Hien T, Reidenbach, Andrew G, Gentile, Juliana E, Kuhn, Eric, O'Moore, Jill, Dooley, Patrick M, Connors, Theresa R, Mazur, Curt, Allen, Shona W, Trombetta, Bianca A, McManus, Alison, Moore, Matthew R, Liu, Jiewu, Cabin, Deborah E, Kordasiewicz, Holly B, Mathews, Joel, Arnold, Steven E, Vallabh, Sonia M, Minikel, Eric Vallabh

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