ReviewNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2010
Strategies for treatment in Alexander disease.
Review in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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
27 citing papers in PubMed, 59 citations in OpenAlex.
- Description of the Hamburg Alexander Leukodystrophy Cohort-Insights into Practical Classification and the Care Situation.Journal of clinical medicine · 2025Article
- Bridging neuro-biomarkers and MR imaging: The synergistic role of glial fibrillary acidic protein in early CNS disease diagnosis.IBRO neuroscience reports · 2025Review
- Misfolding and aggregation in neurodegenerative diseases: protein quality control machinery as potential therapeutic clearance pathways.Cell communication and signaling : CCS · 2024Review
- A retrospective observational cohort study of the anesthetic management and outcomes of pediatric patients with Alexander disease undergoing lumbar puncture or magnetic resonance imaging.Paediatric anaesthesia · 2024Observational
- Alexander disease: the road ahead.Neural regeneration research · 2023Review
- Symptomatic care of late-onset Alexander disease presenting with area postrema-like syndrome with prednisolone; a case report.BMC pediatrics · 2022Article
- Alexander Disease Modeling in Zebrafish: An In Vivo System Suitable to Perform Drug Screening.Genes · 2020Article
- Refining the concept of GFAP toxicity in Alexander disease.Journal of neurodevelopmental disorders · 2019Review
- GFAP Mutations in Astrocytes Impair Oligodendrocyte Progenitor Proliferation and Myelination in an hiPSC Model of Alexander Disease.Cell stem cell · 2018Article
- Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease.Annals of neurology · 2018Article
- Glial fibrillary acidic protein exhibits altered turnover kinetics in a mouse model of Alexander disease.The Journal of biological chemistry · 2017Article
- Modeling Alexander disease with patient iPSCs reveals cellular and molecular pathology of astrocytes.Acta neuropathologica communications · 2016Article
- Stem cell therapy for white matter disorders: don't forget the microenvironment!Journal of inherited metabolic disease · 2016Article
- Astrogliopathology in neurological, neurodevelopmental and psychiatric disorders.Neurobiology of disease · 2016Article
- CSF and Blood Levels of GFAP in Alexander DiseaseeNeuro · 2015Article
- Glial fibrillary acidic protein: from intermediate filament assembly and gliosis to neurobiomarker.Trends in neurosciences · 2015Review
- Lithium Decreases Glial Fibrillary Acidic Protein in a Mouse Model of Alexander Disease.PloS one · 2015Article
- Type 1 equilibrative nucleoside transporter regulates astrocyte-specific glial fibrillary acidic protein expression in the striatum.Brain and behavior · 2014Article
- Protein changes in immunodepleted cerebrospinal fluid from a transgenic mouse model of Alexander disease detected using mass spectrometry.Journal of proteome research · 2013Article
- GFAP expression as an indicator of disease severity in mouse models of Alexander disease.ASN neuro · 2013Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Alexander disease is a rare and generally fatal disorder of the CNS, originally classified among the leukodystrophies because of the prominent myelin deficits found in young patients. The most common form of this disease affects infants, who often have profound mental retardation and a variety of developmental delays, but later onset forms also occur, sometimes with little or no white matter pathology at all. The pathological hallmark of Alexander disease is the inclusion body, known as Rosenthal fiber, within the cell bodies and processes of astrocytes. Recent genetic studies identified heterozygous missense mutations in glial fibrillary acidic protein (GFAP), the major intermediate filament protein in astrocytes, as the cause of nearly all cases of Alexander disease. These studies have transformed our view of this disorder and opened new directions for investigation and clinical practice, particularly with respect to diagnosis. Mechanisms by which expression of mutant forms of glial fibrillary acidic protein (GFAP) lead to the pleiotropic manifestations of disease (afflicting cell types beyond the ones expressing the mutant gene) are slowly coming into focus. Ideas are beginning to emerge that suggest several compelling therapeutic targets for interventions that might slow or arrest the evolution of the disease. This review will outline the rationale for pursuing these strategies, and highlight some of the critical issues that must be addressed in the planning of future clinical trials.
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Registered trials
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.