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Alternative processing of human HTT mRNA with implications for Huntington's disease therapeutics.
Published in:
Brain: A Journal of Neurology, 2022, v. 145, n. 12, p. 4409, doi. 10.1093/brain/awac241
By:
- 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
Publication type:
Article
Results of the first‐in‐human, randomized, double‐blind, placebo‐controlled phase 1b study of lumbar intrathecal bolus administrations of antisense oligonucleotide (ISIS 814907; BIIB080) targeting tau mRNA in patients with mild Alzheimer's disease
Published in:
Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2021, v. 17, p. 1, doi. 10.1002/alz.051871
By:
- Mummery, Catherine J;
- Junge, Candice;
- Kordasiewicz, Holly B;
- Mignon, Laurence;
- Moore, Katrina M;
- Yun, Chris;
- Baumann, Tiffany L;
- Li, Dan;
- Norris, Daniel A;
- Crean, Rebecca;
- Graham, Danielle;
- Huang, Ellen;
- Ratti, Elena;
- Lane, Roger M
Publication type:
Article
DISCOVERY AND EARLY CLINICAL DEVELOPMENT OF IONIS-MAPTRX, THE FIRST TAU-LOWERING ANTISENSE OLIGONUCLEOTIDE, IN PATIENTS WITH MILD AD.
Published in:
Alzheimer's & Dementia: The Journal of the Alzheimer's Association, 2017, v. 13, p. P600, doi. 10.1016/j.jalz.2017.07.242
By:
- Lane, Roger M.;
- Kordasiewicz, Holly B.;
- Smith, Anne;
- Mignon, Laurence;
- Miller, Timothy M.;
- Mummery, Catherine J.;
- Andreasen, Niels;
- Narayanan, Padma;
- Swayze, Eric;
- Norris, Daniel A.;
- Fitzsimmons, Bethany;
- Bennett, C Frank
Publication type:
Article
Characterization of the Prion Protein Binding Properties of Antisense Oligonucleotides.
Published in:
Biomolecules (2218-273X), 2020, v. 10, n. 1, p. 1, doi. 10.3390/biom10010001
By:
- 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.
Publication type:
Article
In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides.
Published in:
Molecular Therapy, 2014, v. 22, n. 12, p. 2093, doi. 10.1038/mt.2014.153
By:
- 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
Publication type:
Article
Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks.
Published in:
JAMA Neurology, 2020, v. 77, n. 6, p. 764, doi. 10.1001/jamaneurol.2020.0299
By:
- Leavitt, Blair R.;
- Kordasiewicz, Holly B.;
- Schobel, Scott A.
Publication type:
Article
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.
Published in:
2018
By:
- McCampbell, Alex;
- Cole, Tracy;
- Wegener, Amy J.;
- Tomassy, Giulio S.;
- Setnicka, Amy;
- Farley, Brandon J.;
- Schoch, Kathleen M.;
- Hoye, Mariah L.;
- Shabsovich, Mark;
- Linhong Sun;
- Yi Luo;
- Mingdi Zhang;
- Sai Thankamony;
- Salzman, David W.;
- Cudkowicz, Merit;
- Graham, Danielle L.;
- Bennett, C. Frank;
- Kordasiewicz, Holly B.;
- Swayze, Eric E.;
- Miller, Timothy M.
Publication type:
journal article
PMP22 antisense oligonucleotides reverse Charcot-Marie-Tooth disease type 1A features in rodent models.
Published in:
2018
By:
- Hien Tran Zhao;
- Damle, Sagar;
- Ikeda-Lee, Karli;
- Kuntz, Steven;
- Jian Li;
- Mohan, Apoorva;
- Kim, Aneeza;
- Hung, Gene;
- Scheideler, Mark A.;
- Scherer, Steven S.;
- Svaren, John;
- Swayze, Eric E.;
- Kordasiewicz, Holly B.;
- Zhao, Hien Tran;
- Li, Jian
Publication type:
journal article
Protein production is an early biomarker for RNA‐targeted therapies.
Published in:
Annals of Clinical & Translational Neurology, 2018, v. 5, n. 12, p. 1492, doi. 10.1002/acn3.657
By:
- 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.
Publication type:
Article
Huntingtin suppression restores cognitive function in a mouse model of Huntington disease.
Published in:
Science Translational Medicine, 2018, v. 10, n. 461, p. 1, doi. 10.1126/scitranslmed.aar3959
By:
- 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.
Publication type:
Article
Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.
Published in:
Science Translational Medicine, 2017, v. 9, n. 374, p. 1, doi. 10.1126/scitranslmed.aag0481
By:
- DeVos, Sarah L.;
- Miller, Rebecca L.;
- Schoch, Kathleen M.;
- Holmes, Brandon B.;
- Kebodeaux, Carey S.;
- Wegener, Amy J.;
- Guo Chen;
- Tao Shen;
- Tran, Hien;
- Nichols, Brandon;
- Zanardi, Tom A.;
- Kordasiewicz, Holly B.;
- Swayze, Eric E.;
- Bennett, C. Frank;
- Diamond, Marc I.;
- Miller, Timothy M.
Publication type:
Article
Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.
Published in:
2018
By:
- 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.
Publication type:
journal article
A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.
Published in:
Human Molecular Genetics, 2017, v. 26, n. 6, p. 1115, doi. 10.1093/hmg/ddx021
By:
- Southwell, Amber L.;
- Skotte, Niels H.;
- Villanueva, Erika B.;
- Østergaard, Michael E.;
- Xiaofeng Gu;
- Kordasiewicz, Holly B.;
- Kay, Chris;
- Cheung, Daphne;
- Yuanyun Xie;
- Waltl, Sabine;
- Dal Cengio, Louisa;
- Findlay-Black, Hailey;
- Doty, Crystal N.;
- Petoukhov, Eugenia;
- Iworima, Diepiriye;
- Slama, Ramy;
- Ooi, Jolene;
- Pouladi, Mahmoud A.;
- Yang, X. William;
- Swayze, Eric E.
Publication type:
Article
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.
Published in:
Human Molecular Genetics, 2016, v. 25, n. R1, p. R53, doi. 10.1093/hmg/ddv442
By:
- Keiser, Megan S.;
- Kordasiewicz, Holly B.;
- McBride, Jodi L.
Publication type:
Article
A single-cell map of antisense oligonucleotide activity in the brain.
Published in:
Nucleic Acids Research, 2023, v. 51, n. 14, p. 7109, doi. 10.1093/nar/gkad371
By:
- 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
Publication type:
Article
MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.
Published in:
Journal of Neuroscience, 2017, v. 37, n. 22, p. 5574, doi. 10.1523/JNEUROSCI.3582-16.2017
By:
- Hoye, Mariah L.;
- Koval, Erica D.;
- Wegener, Amy J.;
- Hyman, Theodore S.;
- Chengran Yang;
- O'Brien, David R.;
- Miller, Rebecca L.;
- Cole, Tracy;
- Schoch, Kathleen M.;
- Tao Shen;
- Tomonori Kunikata;
- Richard, Jean-Philippe;
- Gutmann, David H.;
- Maragakis, Nicholas J.;
- Kordasiewicz, Holly B.;
- Dougherty, Joseph D.;
- Miller, Timothy M.
Publication type:
Article
C-termini of P/Q-type Ca2+ channel α1A subunits translocate to nuclei and promote polyglutamine-mediated toxicity.
Published in:
Human Molecular Genetics, 2006, v. 15, n. 10, p. 1587, doi. 10.1093/hmg/ddl080
By:
- Kordasiewicz, Holly B.;
- Thompson, Randall M.;
- Clark, H. Brent;
- Gomez, Christopher M.
Publication type:
Article
Peripheral huntingtin silencing does not ameliorate central signs of disease in the B6.Htt<sup>Q111/+</sup> mouse model of Huntington’s disease.
Published in:
PLoS ONE, 2017, v. 12, n. 4, p. 1, doi. 10.1371/journal.pone.0175968
By:
- 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.
Publication type:
Article

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