Design of the First-in-Human study of IONIS-MAPTRx, a Tau-lowering antisense oligonucleotide, in patients with alzheimer disease (S2.006)
Abstract
To design a study to test IONIS-MAPTRx an antisense oligonucleotide (ASO) developed to specifically, potently and safely reduce MAPT mRNA in patients with tauopathies.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Anti-sense oligonucleotides to decrease tau expression lead to reversal of tau pathology in mouse and non-human primate models.
“An alternative and promising strategy has focused on using anti-sense oligonucleotides (ASO) to decrease tau expression, leading to reversal of tau pathology in mouse and non-human primate models”
From Targeted degradation of aberrant tau in frontotemporal dementia patient-derived neuronal cell models (2019) · cites this paper as Mignon et al., 2018
Cited by
Sorted by citations
BACE1 inhibitors: Current status and future directions in treating Alzheimer's disease
Moussa‐Pacha, N. M., Abdin, S. M., Omar, H. A., Alniss, H. Y., & Al‐Tel, T. H. (2019) · Medicinal Research Reviews · doi:10.1002/med.21622 · Cited by 308 · see citing works
A walk through tau therapeutic strategies
Jadhav, S., Ávila, J., Schöll, M., Kovács, G. G., Kövari, E., Škrabana, R., Evans, L., Kontseková, E., Malawska, B., de Silva, R., et al. (2019) · Acta Neuropathologica Communications · doi:10.1186/s40478-019-0664-z · Cited by 286 · see citing works
Targeted degradation of aberrant tau in frontotemporal dementia patient-derived neuronal cell models
Silva, M. C., Ferguson, F. M., Cai, Q., Donovan, K. A., Nandi, G., Patnaik, D., Zhang, T., Huang, H.-T., Lucente, D. E., Dickerson, B. C., et al. (2019) · eLife · doi:10.7554/elife.45457 · Cited by 283 · see citing works
Silva, M. C., Nandi, G. A., Tentarelli, S., Gurrell, I. K., Jamier, T., Lucente, D., Dickerson, B. C., Brown, D. G., Brandon, N. J., & Haggarty, S. J. (2020) · Nature Communications · doi:10.1038/s41467-020-16984-1 · Cited by 157 · see citing works
The human motor cortex microcircuit: insights for neurodegenerative disease
McColgan, P., Joubert, J., Tabrizi, S. J., & Rees, G. E. (2020) · Nature reviews. Neuroscience · doi:10.1038/s41583-020-0315-1 · Cited by 86 · see citing works
Tauopathies: Deciphering Disease Mechanisms to Develop Effective Therapies
Silva, M. C., & Haggarty, S. J. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21238948 · Cited by 87 · see citing works
A critical appraisal of tau‐targeting therapies for primary and secondary tauopathies
Imbimbo, B. P., Ippati, S., Watling, M., & Balducci, C. (2021) · Alzheimer s & Dementia · doi:10.1002/alz.12453 · Cited by 72 · see citing works
Therapy for Alzheimer’s disease: Missing targets and functional markers?
Stoiljković, M., Horváth, T. L., & Hajós, M. (2021) · Ageing Research Reviews · doi:10.1016/j.arr.2021.101318 · Cited by 72 · see citing works
Alzheimer’s disease and related tauopathies: disorders of disrupted neuronal identity
Frost, B. E. (2023) · Trends in Neurosciences · doi:10.1016/j.tins.2023.07.006 · Cited by 56 · see citing works
“Current tau-based therapeutic efforts primarily focus on reducing overall tau levels [17,18], preventing tau phosphorylation, or blocking tau spread [19,20].”
Helm, J., Schöls, L. J., & Hauser, S. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14081708 · Cited by 47 · see citing works
Annadurai, N., Sanctis, J. B. D., Hajdúch, M., & Das, V. (2021) · Experimental Neurology · doi:10.1016/j.expneurol.2021.113756 · Cited by 41 · see citing works
Targeting epigenetics: A novel promise for Alzheimer’s disease treatment
Jeremic, D., Jiménez‐Díaz, L., & Navarro‐López, J. D. (2023) · Ageing Research Reviews · doi:10.1016/j.arr.2023.102003 · Cited by 36 · see citing works
Tau; One Protein, So Many Diseases
Tabeshmehr, P., & Eftekharpour, E. (2023) · Biology · doi:10.3390/biology12020244 · Cited by 33 · see citing works
Easton, A. E., Jensen, M. L., Wang, C., Hagedorn, P., Li, Y.-W., Weed, M. R., Meredith, J. E., Guss, V. L., Jones, K. M., Gill, M. B., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.07.027 · Cited by 27 · see citing works
Diligent Design Enables Antibody-ASO Conjugates with Optimal Pharmacokinetic Properties
Sela, T., Mansø, M., Siegel, M., Marban-Doran, C., Ducret, A., Niewöhner, J., Ravn, J., Martin, R. E., Sommer, A., Lohmann, S., et al. (2023) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.3c00393 · Cited by 21 · see citing works
Lozupone, M., Dibello, V., Sardone, R., Castellana, F., Zupo, R., Lampignano, L., Bortone, I., Stallone, R., Altamura, M., Bellomo, A., et al. (2023) · Expert Opinion on Drug Discovery · doi:10.1080/17460441.2023.2200245 · Cited by 19 · see citing works
FTLD Treatment: Current Practice and Future Possibilities
Ljubenkov, P. A., & Boxer, A. L. (2021) · Advances in experimental medicine and biology · doi:10.1007/978-3-030-51140-1_18 · Cited by 13 · see citing works
Kammula, S. V., Tripathi, S., Wang, N., Dawson, V. L., Dawson, T. M., & Mao, X. (2023) · Ageing and Neurodegenerative Diseases · doi:10.20517/and.2023.20 · Cited by 9 · see citing works
The tauopathies: Neuroimaging characteristics and emerging experimental therapies
Riley, K. J., Graner, B. D., & Veronesi, M. C. (2022) · Journal of Neuroimaging · doi:10.1111/jon.13001 · Cited by 7 · see citing works
Alzheimer’s Disease Pharmacology
Plascencia‐Villa, G., & Perry, G. L. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-820472-6.00018-9 · Cited by 6 · see citing works
Emerging drugs for progressive supranuclear palsy
Giagkou, N., & Stamelou, M. (2019) · Expert Opinion on Emerging Drugs · doi:10.1080/14728214.2019.1609450 · Cited by 6 · see citing works
Pasteris, M., Çakır, S., Bellizzi, A., & Sariyer, I. K. (2025) · Journal of Alzheimer s Disease · doi:10.1177/13872877251359633 · Cited by 4 · see citing works
“72 – 74 In addition to ASOs, CRISPR/Cas9-based approaches have shown promise, particularly in correcting the apolipoprotein E ( APOE4 ) genotype to the APOE3 allele in human induced pluripotent stem cells (hiPSCs) derived from AD patients, potentially alleviating the harmful effects of the APOE4…”
Clinical Trial Development in Frontotemporal Lobar Degeneration
Ljubenkov, P. A., & Boxer, A. L. (2022) · Cambridge University Press eBooks · doi:10.1017/9781108975759.019 · Cited by 3 · see citing works
Toward nanomedicine-enabled RNA therapeutics for Alzheimer’s disease
Kara, G., & Villapol, S. (2026) · Molecular Neurodegeneration Advances · doi:10.1186/s44477-026-00026-z · Cited by 3 · see citing works
A state-of-the-art review on miRNA in prevention and treatment of Alzheimer’s disease
Guo, X. (2023) · Journal of Zhejiang University (Medical Sciences) · doi:10.3724/zdxbyxb-2023-0324 · Cited by 2 · see citing works
Nucleic Acid-Based Therapeutics Relevant to Neuroimmune Conditions
Greenfield, A. L., & Hauser, S. L. (2019) · Neurotherapeutics · doi:10.1007/s13311-019-00740-5 · Cited by 2 · see citing works
Vemula, P., Schoch, K. M., & Miller, T. M. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.12.29.522258
“These findings led to the design of the first-in-human study of human tau lowering ASO, IONIS-MAPTRX (Mignon et al., 2018).”
BackgroundSelf-citationStudy of the Neuron-Astrocyte Relationship in a Rodent Model of Tauopathy
de Gérando, A. M. (2019) · theses.fr (ABES) · doi:10.70675/04658671zaca3z4fefza24dz2f5a011bebfe
- Authors
- Mignon, L., Kordasiewicz, H., Lane, R., Smith, A., Miller, T., Narayanan, P., Swayze, E., Norris, D., Fitzsimmons, B., & Bennett, F.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Washington University in St. Louis (US)
- Venue
- Neurology
- Published
- 2018-04-10
- Volume
- 90
- Issue
- 15_supplement
- ISSN
- 0028-3878 (ISSN-L)
- 1526-632X
- OpenAlex
- W2804195724
- Cited by
- 29
- References
- 0
- Citations by year
- 2026: 1 · 2025: 1 · 2023: 7 · 2022: 5 · 2021: 6 · 2020: 3 · 2019: 6
- FWCI
- 2.58
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Neurology (publishedVersion)
- Topics
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Keywords
- antisense oligonucleotides, Alzheimer's disease, tauopathy, tau-targeted therapy
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review