What question did this study set out to answer?

To evaluate tissue distribution, biochemical activity, and histopathological effects of recombinant beta-glucuronidase enzyme replacement therapy in a mouse model of mucopolysaccharidosis type VII (MPS VII).

June 1, 1994Journal of Clinical InvestigationOpen Access

Enzyme replacement therapy for murine mucopolysaccharidosis type VII.

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To evaluate tissue distribution, biochemical activity, and histopathological effects of recombinant beta-glucuronidase enzyme replacement therapy in a mouse model of mucopolysaccharidosis type VII (MPS VII).
Assessed 6-week-old MPS VII mice receiving either a single intravenous injection of 28,000 U recombinant beta-glucuronidase at 5 weeks of age or six weekly injections of 28,000 U starting at birth.
Compared treated mice against untreated 6-week-old MPS VII controls, measuring enzyme distribution and histopathological changes in liver, spleen, kidney, skeleton, and brain.
Mice receiving weekly injections achieved beta-glucuronidase levels of 27.8% in liver, 3.5% in spleen, 3.3% in kidney, and 2.0% to 12.1% in brain relative to normal controls.
Treatment reduced secondary elevations of alpha-galactosidase and beta-hexosaminidase in visceral organs and brain, reduced skeletal pathology, and cleared lysosomal storage in meninges and select neurons, though chondrocytes and glia showed no improvement.

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Implication

Preclinical study demonstrates reduced lysosomal storage in newborn MPS VII mice, indicating early enzyme replacement mitigates disease progression.

Cite This Study

Sands et al. (1994) studied this question.

synapsesocial.com/papers/6a729a93439bab0cabc3e1d4 https://doi.org/10.1172/jci117237

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Implication

Preclinical study demonstrates reduced lysosomal storage in newborn MPS VII mice, indicating early enzyme replacement mitigates disease progression.

Enzyme replacement therapy for murine mucopolysaccharidosis type VII.

Key Points

Discussion

Implication

Cite This Study

Enzyme replacement therapy for murine mucopolysaccharidosis type VII.

Key Points

Discussion

Implication

Cite This Study

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