ArticleScience translational medicine2021
Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.
Abstract read
In one paragraph
Article in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed, 1 pooled it
–field-weighted citation impact
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
37 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Exploiting blood-based biomarkers to align preclinical models with human traumatic brain injury.Brain : a journal of neurology · 2025Pooled it
- Behavioral characterization of bulbar sensorimotor function in a rat model of Alexander disease.Behavioural brain research · 2026Article
- Astrocytes in Neurodegeneration: Spatial States, Crosstalk, and Emerging Therapies.Molecular neurobiology · 2026Review
- Progressive gait and motor deficits in a rat model of Alexander disease.Behavioural brain research · 2026Article
- Characterization of Clinical Phenotype to Glial Fibrillary Acidic Protein Concentrations in Alexander Disease.Annals of clinical and translational neurology · 2026Article
- Synergistic Effects of Glial Fibrillary Acidic Protein Mutation and Overexpression in the Pathogenesis of Alexander Disease.International journal of molecular sciences · 2026Article
- Curing the brain: in search for new astrocyte-specific therapies.Experimental & molecular medicine · 2026Review
- Growth Differentiation Factor 15 Elevation in the Central Nervous System Is Associated With Failure to Thrive in Alexander Disease.Annals of clinical and translational neurology · 2026Article
- Cellular deconvolution of the brain with topological magnetic resonance image analysis.bioRxiv : the preprint server for biology · 2025Article
- Comparison ofMolecular therapy. Nucleic acids · 2025Article
- Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications.Molecular therapy. Nucleic acids · 2025Article
- Alexander's Disease: Potential Drug Targets and Future Directions.Molecular neurobiology · 2025Review
- Article
- Stem cell and gene therapies for leukodystrophies.Molecular therapy. Methods & clinical development · 2025Review
- Self-silencingJournal of neurology, neurosurgery, and psychiatry · 2025Article
- Bilateral vestibulopathy in Alexander disease type II- a case report.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2025Article
- Deletions in Glial Fibrillary Acidic Protein Leading to Alterations in Intermediate Filament Assembly and Network Formation.International journal of molecular sciences · 2025Article
- Article
- Astrocytes in Primary Familial Brain Calcification (PFBC): Emphasis on the Importance of Induced Pluripotent Stem Cell-Derived Human Astrocyte Models.Advances in experimental medicine and biology · 2025Review
- Morphological Characteristics and Extracellular Matrix Abnormalities in Astrocytes Derived From iPSCs of Children With Alexander Disease.CNS neuroscience & therapeutics · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors.
Tracy L HagemannWaisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.ORCID 0000-0002-0237-9267
Berit PowersIonis Pharmaceuticals, Carlsbad, CA 92010, USA.
Ni-Hsuan LinInstitute of Molecular Medicine, College of Life Sciences, National Tsing Hua University, Hsinchu 30013, Taiwan.ORCID 0000-0003-4524-3615
Ahmed F MohamedWaisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
Katerina L DagueWaisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.ORCID 0000-0001-5613-9018
Seth C HannahWaisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
Gemma BachmannIonis Pharmaceuticals, Carlsbad, CA 92010, USA.ORCID 0000-0002-3974-191X
Curt MazurIonis Pharmaceuticals, Carlsbad, CA 92010, USA.ORCID 0000-0003-3485-9086
Frank RigoIonis Pharmaceuticals, Carlsbad, CA 92010, USA.
Abby L OlsenDepartment of Neurology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0002-7680-1758
Mel B FeanyDepartment of Pathology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.ORCID 0000-0003-0315-7970
Ming-Der PerngInstitute of Molecular Medicine, College of Life Sciences, National Tsing Hua University, Hsinchu 30013, Taiwan.ORCID 0000-0002-1884-3714
Robert F BermanDepartment of Neurological Surgery and M.I.N.D Institute, University of California, Davis, Davis, CA 95616, USA.ORCID 0000-0001-8530-3147
Albee MessingWaisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.ORCID 0000-0001-6049-0521
Funding
WISCONSIN CENTER ON MENTAL RETARDATION: CORE SUPPORTP30HD003352 · NICHD · UNIVERSITY OF WISCONSIN-MADISON · PI DAVIDSON, RICHARD J · 1985 to 2015
$25.2MResearch Project: Pathologic Significance of Maternal AutoantibodiesP50HD103526 · NICHD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI LEONARD J. ABBEDUTO, Melissa Dawn Bauman · 2020 to 2026
$9.7MWaisman Intellectual and Developmental Disabilities Research Center Down Syndrome RegistryU54HD090256 · NICHD · UNIVERSITY OF WISCONSIN-MADISON · PI CHANG, QIANG · 2016 to 2020
$6.0MPatient-derived iPS cells as model systems for AxDP01HD076892 · NICHD · UNIVERSITY OF WISCONSIN-MADISON · PI ZHANG, SU-CHUN · 2014 to 2018
$6.0MAstrocyte-synapse interactions in a rat model of Alexander diseaseR01NS110719 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI HAGEMANN, TRACY · 2019 to 2023
$2.1MDefining the genetic contribution of glia in Parkinson's disease pathogenesisK08NS109344 · NINDS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI OLSEN, ABBY LAUREN · 2018 to 2022
$997kNICHD NIH HHS P01 HD076892NICHD NIH HHS P30 HD003352NICHD NIH HHS P50 HD103526NICHD NIH HHS U54 HD090256NINDS NIH HHS K08 NS109344NINDS NIH HHS R01 NS110719
Abstract
Alexander disease (AxD) is a devastating leukodystrophy caused by gain-of-function mutations in
Indexed as
Alexander DiseaseGlial Fibrillary Acidic ProteinMotor DisordersWhite MatterAnimalsAstrocytesGliosisMutationRatsGfap protein, ratGlial Fibrillary Acidic Protein
Identifiers
What OpenQuestion holds
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Read underepoch 390
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.