ArticleGene therapy2006
Embryonic stem cell-based reduction of central nervous system sulfatide storage in an animal model of metachromatic leukodystrophy.
Article in Gene therapy, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 37 citations in OpenAlex.
- Mesenchymal Stem Cell-Based Therapy for Lysosomal Storage Diseases and Other Neurodegenerative Disorders.Frontiers in pharmacology · 2022Review
- Sphingolipid lysosomal storage diseases: from bench to bedside.Lipids in health and disease · 2021Review
- Progression-Related Loss of Stromal Caveolin 1 Levels Mediates Radiation Resistance in Prostate Carcinoma via the Apoptosis Inhibitor TRIAP1.Journal of clinical medicine · 2019Article
- 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
- Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Review
- Developing therapeutic approaches for metachromatic leukodystrophy.Drug design, development and therapy · 2013Review
- Pathology and current treatment of neurodegenerative sphingolipidoses.Neuromolecular medicine · 2010Review
- Multi-system disorders of glycosphingolipid and ganglioside metabolism.Journal of lipid research · 2010Review
- Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease.Stem cells (Dayton, Ohio) · 2009Article
- Lysosulfatide regulates the motility of a neural precursor cell line via calcium-mediated process collapse.Neurochemical research · 2009Article
- Enzyme, cell and gene-based therapies for metachromatic leukodystrophy.Journal of inherited metabolic disease · 2007Review
- Article
- Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer.Gene therapy · 2007Article
- The role and metabolism of sulfatide in the nervous system.Molecular neurobiologyReview
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pluripotency, virtually unlimited self-renewal and amenability to genetic modification make embryonic stem (ES) cells an attractive donor source for cell-mediated gene therapy. In this proof of concept study, we explore whether glial precursors derived from murine ES cells (ESGPs) and engineered to overexpress human arylsulfatase A (hASA) can cross-correct the metabolic defect in an animal model of metachromatic leukodystrophy (MLD). Transfected ES cells showed an up to 30-fold increase in ASA activity. Following in vitro differentiation, high expression of ASA was found in all stages of neural and glial differentiation. hASA-overexpressing ESGPs maintained their ability to differentiate into astrocytes and oligodendrocytes in vitro and in vivo. After transplantation into the brain of neonatal ASA-deficient mice, hASA-overexpressing ESGPs were found to incorporate into a variety of host brain regions. Four weeks after engraftment, immunofluorescence analyses with an antibody to sulfatide revealed a 46.7+/-4.0% reduction of immunoreactive sulfatide deposits in the vicinity of the hASA-positive engrafted cells, thereby significantly extending the rate of sulfatide reduction achieved by the endogenous ASA activity of non-hASA-transfected control cells (21.1+/-5.8%). These findings provide first in vivo evidence that ES cells may serve as a potential donor source for cell-mediated enzyme delivery in storage disorders such as MLD.
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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.