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Blood, 1 March 2001, Vol. 97, No. 5, pp. 1498-1504
TRANSPLANTATION
Nonablative neonatal marrow transplantation attenuates functional
and physical defects of  -glucuronidase deficiency
Brian W. Soper,
Mark D. Lessard,
Carole A. Vogler,
Beth Levy,
Wesley G. Beamer,
William S. Sly, and
Jane E. Barker
From The Jackson Laboratory, Bar Harbor, ME; Department
of Pathology, Saint Louis University School of Medicine, St Louis, MO;
and the Edward A. Doisy Department of Biochemistry and Molecular
Biology, Saint Louis University School of Medicine, St Louis, MO.
The toxicity of preparative regimens render neonatal bone marrow
transplantation (BMT) for progressive childhood diseases a
controversial treatment. Ablative BMT in neonatal mice with or without
the lysosomal storage disease mucopolysaccharidosis type VII (MPS VII)
show high morbidity and developmental disruption of both brain and bone
structure. In this investigation, BMT was performed with a high dose of
congenic, normal bone marrow into nonablated newborn mice. Recipients
had lifelong, multilineage, peripheral blood chimerism with the donor
 -glucuronidase-positive (GUS+) cells that was both well
tolerated and therapeutic. Three daily injections of normal adult
marrow increased the average life span by at least 6 months and
corrected the functional breeding deficits typical of the MPS VII mice.
Twelve months after injection, several structural features of femurs
were more like that of normal mice than of untreated MPS VII mice.
Periosteal circumference and bone cortical thickness were significantly
improved in males and cortical density did not differ significantly
from values in normal females. Significant reduction of lysosomal
glycosaminoglycan storage corresponded directly with GUS enzyme
activity and percentage of histochemically GUS+ cells in
visceral organs and hematopoietic tissues such as thymus, spleen,
peripheral blood, and bone marrow. By all criteria tested, BMT into
neonatal MPS VII mice in the absence of any preparative regimen is a
successful therapy.
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