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Blood, Vol. 95 No. 11 (June 1), 2000:
pp. 3631-3633
BRIEF REPORT
Reduction of lysosomal storage in murine mucopolysaccharidosis
type VII by transplantation of normal and genetically modified
macrophages
Toya Ohashi,
Takashi Yokoo,
Sayoko Iizuka,
Hiroshi Kobayashi,
William S. Sly, and
Yoshikatsu Eto
From the Department of Gene Therapy, Institute of DNA
Medicine, Jikei University School of Medicine, Tokyo, Japan;
Department of Pediatrics, Jikei University School of Medicine, Tokyo,
Japan; Department of Medicine II, Jikei University School of Medicine,
Tokyo, Japan; Edward A. Doisy Department of Medicine/Cardiology, and
Molecular Biology, St Louis University School of Medicine, St
Louis, MO.
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  -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.
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