Kordasiewicz, Holly

Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43

Polymenidou, M., Lagier-Tourenne, C., Hutt, K. R., Huelga, S. C., Moran, J., Liang, T. Y., Ling, S.-C., Sun, E., Wancewicz, E., Mazur, C., et al. (2011) · Nature Neuroscience

Amyotrophic Lateral Sclerosis ResearchRNA Research and Splicing

Cited by 1,335

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

Kordasiewicz, H. B., Stanek, L. M., Wancewicz, E. V., Mazur, C., McAlonis, M. M., Pytel, K. A., Artates, J. W., Weiss, A., Cheng, S. H., Shihabuddin, L. S., et al. (2012) · Neuron

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 770

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy

DeVos, S. L., Miller, R. L., Schoch, K. M., Holmes, B. B., Kebodeaux, C. S., Wegener, A. J., Chen, G., Shen, T., Tran, H., Nichols, B., et al. (2017) · Science Translational Medicine

Alzheimer's disease research and treatmentsAmyotrophic Lateral Sclerosis Research

Cited by 516

Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial

Mummery, C. J., Börjesson-Hanson, A., Blackburn, D. J., Vijverberg, E. G. B., De Deyn, P. P., Ducharme, S., Jonsson, M., Schneider, A., Rinne, J. O., Ludolph, A. C., et al. (2023) · Nature Medicine

Alzheimer's disease research and treatmentsDementia and Cognitive Impairment Research

Cited by 347

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

McCampbell, A., Cole, T., Wegener, A. J., Tomassy, G. S., Setnicka, A., Farley, B. J., Schoch, K. M., Hoye, M. L., Shabsovich, M., Sun, L., et al. (2018) · Journal of Clinical Investigation

Amyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Research

Cited by 279

The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Jafar-nejad, P., Powers, B., Soriano, A., Zhao, H., Norris, D. A., Matson, J., DeBrosse-Serra, B., Watson, J., Narayanan, P., Chun, S. J., et al. (2021) · Nucleic Acids Research

RNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Cited by 128

Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease

Southwell, A. L., Kordasiewicz, H. B., Langbehn, D., Skotte, N. H., Parsons, M. P., Villanueva, E. B., Caron, N. S., Østergaard, M. E., Anderson, L. M., Xie, Y., et al. (2018) · Science Translational Medicine

Genetic Neurodegenerative DiseasesAmyotrophic Lateral Sclerosis Research

Cited by 126

Antisense oligonucleotide–mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles

Friedrich, J., Kordasiewicz, H. B., O’Callaghan, B., Handler, H. P., Wagener, C., Duvick, L., Swayze, E. E., Rainwater, O., Hofstra, B., Benneyworth, M., et al. (2018) · JCI Insight

Genetic Neurodegenerative DiseasesAmyotrophic Lateral Sclerosis Research

Cited by 121

Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging

Mazur, C., Powers, B., Zasadny, K., Sullivan, J. M., Dimant, H., Kamme, F., Hesterman, J., Matson, J., Oestergaard, M., Seaman, M., et al. (2019) · JCI Insight

RNA Interference and Gene DeliveryNeurogenetic and Muscular Disorders Research

Cited by 114

Exploratory Tau Biomarker Results From a Multiple Ascending-Dose Study of BIIB080 in Alzheimer Disease: A Randomized Clinical Trial

Edwards, A. L., Collins, J. A., Junge, C., Kordasiewicz, H., Mignon, L., Wu, S., Li, Y., Lin, L., DuBois, J., Hutchison, R. M., et al. (2023) · JAMA Neurology

Alzheimer's disease research and treatmentsDementia and Cognitive Impairment Research

Cited by 113

Antisense drugs make sense for neurological diseases

Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W. (2021) · Annual Review of Pharmacology and Toxicology

RNA Interference and Gene DeliveryAmyotrophic Lateral Sclerosis Research

Cited by 108

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models

Hoye, M. L., Koval, E. D., Wegener, A. J., Hyman, T. S., Yang, C., O'Brien, D. R., Miller, R. L., Cole, T., Schoch, K. M., Shen, T., et al. (2017) · The Journal of Neuroscience

Amyotrophic Lateral Sclerosis ResearchMicroRNA in disease regulation

Cited by 82

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

Mortberg, M. A., Gentile, J. E., Nadaf, N. M., Vanderburg, C., Simmons, S., Dubinsky, D., Slamin, A., Maldonado, S., Petersen, C. L., Jones, N., et al. (2023) · Nucleic Acids Research

RNA Interference and Gene DeliveryAlzheimer's disease research and treatments

Cited by 54

Design of the First-in-Human study of IONIS-MAPTRx, a Tau-lowering antisense oligonucleotide, in patients with alzheimer disease (S2.006)

Mignon, L., Kordasiewicz, H., Lane, R., Smith, A., Miller, T., Narayanan, P., Swayze, E., Norris, D., Fitzsimmons, B., & Bennett, F. (2018) · Neurology

Alzheimer's disease research and treatmentsDementia and Cognitive Impairment Research

Cited by 29

Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002)

Tabrizi, S., Leavitt, B., Kordasiewicz, H., Czech, C., Swayze, E., Norris, D. A., Baumann, T., Gerlach, I., Schobel, S., Smith, A., et al. (2018) · Neurology

Genetic Neurodegenerative DiseasesAmyotrophic Lateral Sclerosis Research

Cited by 26

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

Mummery, C. J., Junge, C., Kordasiewicz, H. B., Mignon, L., Moore, K. M., Yun, C., Baumann, T. L., Li, D., Norris, D. A., Crean, R., et al. (2021) · Alzheimer's & Dementia

Alzheimer's disease research and treatmentsDementia and Cognitive Impairment Research

Cited by 16