Document Detail

Reduction of lysosomal storage in murine mucopolysaccharidosis type VII by transplantation of normal and genetically modified macrophages.
MedLine Citation:
PMID:  10828055     Owner:  NLM     Status:  MEDLINE    
This study examined the ability of macrophages to serve as target cells of gene therapy for mucopolysaccharidosis (MPS) type VII using a murine model. Bone marrow cells were harvested from syngeneic normal mice and differentiated to macrophages. These cells were given to nonmyeloablated MPS VII mice. After transplantation, donor cells populated the liver and spleen. The pathologic improvement at day 38 after transplantation was significant and glycosaminoglycan storage was reduced. To develop gene therapy using this system, a retroviral vector expressing human beta-glucuronidase (HBG) was used to infect macrophages cultivated from MPS VII mice and given to nonmyeloablated MPS VII mice. At 38 days after transplantation, HBG-positive cells were still observed histochemically and pathologic improvement was significant. These observations suggest that macrophage transplantation is a promising method for treatment of murine MPS VII without myeloablation, and macrophages may be good target cells for ex vivo gene therapy for MPS VII.
T Ohashi; T Yokoo; S Iizuka; H Kobayashi; W S Sly; Y Eto
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Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Blood     Volume:  95     ISSN:  0006-4971     ISO Abbreviation:  Blood     Publication Date:  2000 Jun 
Date Detail:
Created Date:  2000-06-29     Completed Date:  2000-06-29     Revised Date:  2006-11-15    
Medline Journal Info:
Nlm Unique ID:  7603509     Medline TA:  Blood     Country:  UNITED STATES    
Other Details:
Languages:  eng     Pagination:  3631-3     Citation Subset:  AIM; IM    
Department of Gene Therapy, Institute of DNA Medicine, Jikei University School of Medicine, Tokyo, Japan.
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MeSH Terms
Bone Marrow Cells / cytology*
Cell Differentiation
Disease Models, Animal
Gene Therapy*
Glucuronidase / biosynthesis,  deficiency,  genetics*
Kupffer Cells / pathology,  ultrastructure
Lysosomes / pathology,  ultrastructure*
Macrophages / cytology,  transplantation*
Mice, Inbred Strains
Mice, Knockout
Mucopolysaccharidosis VII / therapy*
Recombinant Proteins / biosynthesis
Transplantation, Isogeneic
Reg. No./Substance:
0/Recombinant Proteins; EC

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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