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Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle.
Pediatr Res. 1988 Jul; 24(1):90-4.PR

Abstract

Attempts at treatment of glycogenosis type II and other lysosomal storage disorders by enzyme replacement have been reported. Parenteral enzyme administration has been ineffectual. Treatment by bone marrow transplantation is currently under investigation. We have used cultured skeletal muscle cells from a patient with infantile glycogenosis type II to study fundamental aspects of enzyme replacement therapy. Efficient uptake of acid alpha-glucosidase was achieved by using the mannose-6-phosphate receptor on the cell surface as a target for an enzyme precursor with phosphorylated high-mannose types carbohydrate chains purified from human urine. We found that the enzyme was channeled to the lysosomes and converted to mature acid alpha-glucosidase. Glycogen storage was reversed. The results are discussed in relation to treatment of glycogenosis type II.

Authors+Show Affiliations

Department of Cell Biology and Genetics, Erasmus University, Rotterdam, The Netherlands.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

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

Language

eng

PubMed ID

2970619

Citation

Van der Ploeg, A T., et al. "Receptor-mediated Uptake of Acid Alpha-glucosidase Corrects Lysosomal Glycogen Storage in Cultured Skeletal Muscle." Pediatric Research, vol. 24, no. 1, 1988, pp. 90-4.
Van der Ploeg AT, Loonen MC, Bolhuis PA, et al. Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle. Pediatr Res. 1988;24(1):90-4.
Van der Ploeg, A. T., Loonen, M. C., Bolhuis, P. A., Busch, H. M., Reuser, A. J., & Galjaard, H. (1988). Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle. Pediatric Research, 24(1), 90-4.
Van der Ploeg AT, et al. Receptor-mediated Uptake of Acid Alpha-glucosidase Corrects Lysosomal Glycogen Storage in Cultured Skeletal Muscle. Pediatr Res. 1988;24(1):90-4. PubMed PMID: 2970619.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle. AU - Van der Ploeg,A T, AU - Loonen,M C, AU - Bolhuis,P A, AU - Busch,H M, AU - Reuser,A J, AU - Galjaard,H, PY - 1988/7/1/pubmed PY - 1988/7/1/medline PY - 1988/7/1/entrez SP - 90 EP - 4 JF - Pediatric research JO - Pediatr. Res. VL - 24 IS - 1 N2 - Attempts at treatment of glycogenosis type II and other lysosomal storage disorders by enzyme replacement have been reported. Parenteral enzyme administration has been ineffectual. Treatment by bone marrow transplantation is currently under investigation. We have used cultured skeletal muscle cells from a patient with infantile glycogenosis type II to study fundamental aspects of enzyme replacement therapy. Efficient uptake of acid alpha-glucosidase was achieved by using the mannose-6-phosphate receptor on the cell surface as a target for an enzyme precursor with phosphorylated high-mannose types carbohydrate chains purified from human urine. We found that the enzyme was channeled to the lysosomes and converted to mature acid alpha-glucosidase. Glycogen storage was reversed. The results are discussed in relation to treatment of glycogenosis type II. SN - 0031-3998 UR - https://www.unboundmedicine.com/medline/citation/2970619/Receptor_mediated_uptake_of_acid_alpha_glucosidase_corrects_lysosomal_glycogen_storage_in_cultured_skeletal_muscle_ L2 - http://dx.doi.org/10.1203/00006450-198807000-00021 DB - PRIME DP - Unbound Medicine ER -