ArticleJournal of neuroinflammation2010
Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response.
Article in Journal of neuroinflammation, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 40 citations in OpenAlex.
- Anakinra in Sanfilippo syndrome: a phase 1/2 trial.Nature medicine · 2024Trial
- Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease.Disease models & mechanisms · 2026Article
- Expanding the Spectrum: A Rare Case of Morquio Syndrome With Bronchial Asthma and Seizure Disorder.Clinical case reports · 2025Article
- Femoral Structure and Biomechanical Characteristics in Sanfilippo Syndrome Type-B Mice.International journal of molecular sciences · 2023Article
- Novel therapies for mucopolysaccharidosis type III.Journal of inherited metabolic disease · 2021Review
- Molecular Bases of Neurodegeneration and Cognitive Decline, the Major Burden of Sanfilippo Disease.Journal of clinical medicine · 2020Review
- From Lysosomal Storage Diseases to NKT Cell Activation and Back.International journal of molecular sciences · 2017Review
- Timing of Gene Therapy Interventions: The Earlier, the Better.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.Orphanet journal of rare diseases · 2016Review
- Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I.PloS one · 2016Article
- Manipulating autophagic processes in autoimmune diseases: a special focus on modulating chaperone-mediated autophagy, an emerging therapeutic target.Frontiers in immunology · 2015Review
- Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Altered distribution and function of natural killer cells in murine and human Niemann-Pick disease type C1.Blood · 2014Article
- Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity.Journal of inherited metabolic disease · 2014Review
- Clinical neurogenetics: neuropathic lysosomal storage disorders.Neurologic clinics · 2013Review
- Blood-brain barrier structure and function and the challenges for CNS drug delivery.Journal of inherited metabolic disease · 2013Review
- Immunologic privilege in the central nervous system and the blood-brain barrier.Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2013Review
- hsp70 and a novel axis of type I interferon-dependent antiviral immunity in the measles virus-infected brain.Journal of virology · 2013Article
- Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB.PloS one · 2012Article
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundRecently, using a mouse model of mucopolysaccharidosis (MPS) IIIB, a lysosomal storage disease with severe neurological deterioration, we showed that MPS IIIB neuropathology is accompanied by a robust neuroinflammatory response of unknown consequence. This study was to assess whether MPS IIIB lymphocytes are pathogenic.
methodsLymphocytes from MPS IIIB mice were adoptively transferred to naïve wild-type mice. The recipient animals were then evaluated for signs of disease and inflammation in the central nervous system.
resultsOur results show for the first time, that lymphocytes isolated from MPS IIIB mice caused a mild paralytic disease when they were injected systemically into naïve wild-type mice. This disease is characterized by mild tail and lower trunk weakness with delayed weight gain. The MPS IIIB lymphocytes also trigger neuroinflammation within the CNS of recipient mice characterized by an increase in transcripts of IL2, IL4, IL5, IL17, TNFalpha, IFNalpha and Ifi30, and intraparenchymal lymphocyte infiltration.
conclusionsOur data suggest that an autoimmune response directed at CNS components contributes to MPS IIIB neuropathology independent of lysosomal storage pathology. Adoptive transfer of purified T-cells will be needed in future studies to identify specific effector T-cells in MPS IIIB neuroimmune pathogenesis.
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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.