ArticleStem cells (Dayton, Ohio)2011
Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.
Article in Stem cells (Dayton, Ohio), 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers.
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Who cites it
39 citing papers in PubMed, 67 citations in OpenAlex.
- Chimeric enzymes enhance treatment potential for globoid cell leukodystrophy through hematopoietic stem cell gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Induced Pluripotent Stem Cells for the Treatment of Lysosomal Storage Disorders.Journal of inherited metabolic disease · 2025Review
- Advances in Genetic Reprogramming: Prospects from Developmental Biology to Regenerative Medicine.Current medicinal chemistry · 2024Review
- Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases.Neural regeneration research · 2023Article
- Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Krabbe disease: A personal perspective and hypothesis.BioImpacts : BI · 2022Review
- Isolation and Culture of Neural Stem/Progenitor Cells from the Postnatal Periventricular Region.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Review
- Harnessing the Potential of Stem Cells for Disease Modeling: Progress and Promises.Journal of personalized medicine · 2020Review
- Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Article
- Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Insight into Mechanobiology: How Stem Cells Feel Mechanical Forces and Orchestrate Biological Functions.International journal of molecular sciences · 2019Review
- Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy.Cell death & disease · 2018Article
- A WNT protein therapeutic improves the bone-forming capacity of autografts from aged animals.Scientific reports · 2018Article
- Two factor-based reprogramming of rodent and human fibroblasts into Schwann cells.Nature communications · 2017Article
- Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.Stem cells translational medicine · 2017Article
- Cellular transplant therapies for globoid cell leukodystrophy: Preclinical and clinical observations.Journal of neuroscience research · 2016Review
- Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases.Human gene therapy · 2016Review
- Glial progenitor cell-based treatment of the childhood leukodystrophies.Experimental neurology · 2016Review
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9 authors at 3 institutions in 1 country.
Funding
Abstract
Murine neural stem cells (mNSCs), either naive or genetically modified to express supranormal levels of β-galactocerebrosidase (GALC), were transplanted into the brain of Twitcher mice, a murine model of globoid cell leukodystrophy, a severe sphingolipidosis. Cells engrafted long-term into the host cytoarchitecture, producing functional GALC. Levels of enzyme activity in brain and spinal cord tissues were enhanced when GALC-overexpressing NSC were used. Enzymatic correction correlated with reduced tissue storage, decreased activation of astroglia and microglia, delayed onset of symptoms, and longer lifespan. Mechanisms underlying the therapeutic effect of mNSC included widespread enzyme distribution, cross-correction of host cells, anti-inflammatory activity, and neuroprotection. Similar cell engraftment and metabolic correction were reproduced using human NSC. Thus, NSC gene therapy rapidly reconstitutes sustained and long-lasting enzyme activity in central nervous system tissues. Combining this approach with treatments targeting the systemic disease associated with leukodystrophies may provide significant therapeutic benefit.
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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.