Pediatr Res. 1990 Oct;28(4):344-7.

Rat heart perfusion as model system for enzyme replacement therapy in glycogenosis type II.

van der Ploeg AT, van der Kraaij AM, Willemsen R, Kroos MA, Loonen MC, Koster JF, Reuser AJ.

Source

Sophia Children's Hospital, Rotterdam, The Netherlands.

Abstract

Cardiac failure and skeletal muscle weakness are the main clinical features of glycogenosis type II, a lysosomal storage disorder caused by acid alpha-glucosidase deficiency. In our study, we have investigated in a rat heart perfusion-recirculation system whether acid alpha-glucosidase can be taken up from the vascular system into cardiomyocytes. When rat hearts were perfused with mannose 6-phosphate-containing acid alpha-glucosidase purified from bovine testis, a 3- to 4-fold increase of enzyme activity was obtained. Perfusion with human placental acid alpha-glucosidase not containing the mannose 6-phosphate recognition marker did not have such an effect. The presence of bovine testis acid alpha-glucosidase in heart tissue was demonstrated by immunoblotting. Immunocytochemistry provides evidence for uptake of the exogenous enzyme in lysosomes of the cardiomyocytes. The relevance of these findings for enzyme therapy in glycogenosis type II is discussed.

PMID:: 2235132; [PubMed - indexed for MEDLINE]

Publication Types, MeSH Terms, Substances

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Research Support, Non-U.S. Gov't

MeSH Terms

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alpha-Glucosidases

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Related citations in PubMed

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Cited by 1 PubMed Central article

Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice. [J Clin Invest. 1991]
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