Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

por 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.
Publicado 2017

Characterization of the Prion Protein Binding Properties of Antisense Oligonucleotides

por Reidenbach, Andrew G., Minikel, Eric Vallabh, Zhao, Hien T., Guzman, Stacy G., Leed, Alison J., Mesleh, Michael F., Kordasiewicz, Holly B., Schreiber, Stuart L., Vallabh, Sonia M.
Publicado 2019

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

por 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.
Publicado 2017

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

por 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.
Publicado 2021

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

por 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.
Publicado 2021

Protein production is an early biomarker for RNA‐targeted therapies

por Self, Wade K., Schoch, Kathleen M., Alex, Jacob, Barthélemy, Nicolas, Bollinger, James G., Sato, Chihiro, Cole, Tracy, Kordasiewicz, Holly B., Swayze, Eric, Bateman, Randall J., Miller, Timothy M.
Publicado 2018

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

por 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
Publicado 2023

RNA-targeted therapy corrects neuronal deficits in PACS1 syndrome mice

por 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
Publicado 2023

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

por Mortberg, Meredith A, Gentile, Juliana E, Nadaf, Naeem, 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
Publicado 2023

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

por 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
Publicado 2023

Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment

por 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.
Publicado 2020

Alternative processing of human HTT mRNA with implications for Huntington’s disease therapeutics

por 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
Publicado 2022

Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.Htt(Q111/+) mouse model of Huntington’s disease

por 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.
Publicado 2017

An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement

por Poplawski, Shane G., Garbett, Krassimira A., McMahan, Rebekah L., Kordasiewicz, Holly B., Zhao, Hien, Kennedy, Andrew J., Goleva, Slavina B., Sanders, Teresa H., Motley, S. Timothy, Swayze, Eric E., Ecker, David J., Sweatt, J. David, Michael, Todd P., Greer, Celeste B.
Publicado 2020

Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNS

por 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
Publicado 2022

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

por 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
Publicado 2020

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

por 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.
Publicado 2021