Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.
MS Sands, JE Barker, C Vogler, B Levy… - … ; a journal of technical …, 1993 - europepmc.org
MS Sands, JE Barker, C Vogler, B Levy, B Gwynn, N Galvin, WS Sly, E Birkenmeier
Laboratory investigation; a journal of technical methods and pathology, 1993•europepmc.orgBackground Bone marrow transplantation (BMT) proved an effective therapy for murine
mucopolysaccharidosis type VII (MPS VII) in adult gusmps/gusmps mice with well developed
clinical and pathologic characteristics of the disease. MPS VII mice transplanted as adults
had a marked decrease in lysosomal storage material in many organs, although not in the
skeleton and brain (1). Since untreated newborn MPS VII mice appear normal and have
minimal lysosomal storage material detectable microscopically, we postulated that BMT in …
mucopolysaccharidosis type VII (MPS VII) in adult gusmps/gusmps mice with well developed
clinical and pathologic characteristics of the disease. MPS VII mice transplanted as adults
had a marked decrease in lysosomal storage material in many organs, although not in the
skeleton and brain (1). Since untreated newborn MPS VII mice appear normal and have
minimal lysosomal storage material detectable microscopically, we postulated that BMT in …
Background
Bone marrow transplantation (BMT) proved an effective therapy for murine mucopolysaccharidosis type VII (MPS VII) in adult gusmps/gusmps mice with well developed clinical and pathologic characteristics of the disease. MPS VII mice transplanted as adults had a marked decrease in lysosomal storage material in many organs, although not in the skeleton and brain (1). Since untreated newborn MPS VII mice appear normal and have minimal lysosomal storage material detectable microscopically, we postulated that BMT in newborn mice might prevent the subsequent accumulation of storage material.
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