ArticleGene therapy2000
Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector.
Article in Gene therapy, 2000. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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.
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Who cites it
26 citing papers in PubMed, 66 citations in OpenAlex.
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice.Frontiers in molecular neuroscience · 2021Article
- Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Gene therapy for metachromatic leukodystrophy.Journal of neuroscience research · 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
- Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.Human gene therapy. Clinical development · 2014Article
- Comparison of five peptide vectors for improved brain delivery of the lysosomal enzyme arylsulfatase A.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2014Article
- Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.Human gene therapy · 2012Article
- Successful treatment of metachromatic leukodystrophy using bone marrow transplantation of HoxB4 overexpressing cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Enzyme, cell and gene-based therapies for metachromatic leukodystrophy.Journal of inherited metabolic disease · 2007Review
- Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer.Gene therapy · 2007Article
- Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.The Journal of clinical investigation · 2006Article
- Microglia: a cellular vehicle for CNS gene therapy.The Journal of clinical investigation · 2006Article
- Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2006Article
- Potential of mesenchymal stem cells in gene therapy approaches for inherited and acquired diseases.Expert opinion on biological therapy · 2005Review
- Article
- Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.The Journal of clinical investigation · 2004Article
- Transduction of bone-marrow-derived mesenchymal stem cells by using lentivirus vectors pseudotyped with modified RD114 envelope glycoproteins.Journal of virology · 2004Article
- Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.Gene therapy · 2003Article
Corrections and comments
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Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A deficiency of arylsulfatase A (ASA) results in the lysosomal lipid storage disease metachromatic leukodystrophy. The disease mainly affects the central nervous system causing a progressive demyelination. A therapeutic effect will depend on the delivery of the deficient enzyme to the central nervous system. We have transplanted ASA-deficient mice with bone marrow transduced with a retroviral vector expressing the human ASA cDNA. All transplanted animals initially showed high serum levels of human ASA. In 50% of the recipients high ASA serum levels were sustained for 12 months after transplantation. In the remaining mice, serum levels decreased rapidly to low or undetectable levels. ASA activity and immunoreactivity was detectable in all organs of animals with continuous levels of ASA in serum. Most notably, substantial amounts of ASA activity were transferred into the brain, reaching up to 33% of the normal tissue level. In contrast to peripheral organs, the amount of enzyme delivered to the brain did not correlate with ASA serum levels as an indicator of overexpression. This reveals that enzyme transfer to the brain is not due to endocytosis of serum ASA by endothelial cells, but rather to bone marrow-derived cells migrated into the brain. Gene Therapy (2000) 7, 1250-1257.
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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.