Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system

• Published in: Plant biotechnology journal Vol. 5; no. 5; pp. 579 - 590
• Main Authors: Shaaltiel, Yoseph, Bartfeld, Daniel, Hashmueli, Sharon, Baum, Gideon, Brill-Almon, Einat, Galili, Gad, Dym, Orly, Boldin-Adamsky, Swetlana A., Silman, Israel, Sussman, Joel L., Futerman, Anthony H., Aviezer, David
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.09.2007
• Subjects: Animals; Blotting, Western; cell culture; Cells, Cultured; CHO Cells; Cricetinae; Cricetulus; Crystallography, X-Ray; Daucus carota - cytology; Daucus carota - enzymology; Daucus carota - metabolism; Drug Evaluation, Preclinical - methods; Female; Gaucher Disease - drug therapy; Gaucher Disease - enzymology; Gaucher Disease - genetics; Gaucher's disease; glucocerebrosidase; Glucosylceramidase - genetics; Glucosylceramidase - metabolism; Glucosylceramidase - therapeutic use; Humans; Macrophages - metabolism; Male; mannose; Mannose - chemistry; Mannose - metabolism; Mice; Mice, Inbred ICR; plant; Polysaccharides - chemistry; Polysaccharides - metabolism; Recombinant Proteins - chemistry; Recombinant Proteins - metabolism; Recombinant Proteins - therapeutic use

Summary Gaucher's disease, a lysosomal storage disorder caused by mutations in the gene encoding glucocerebrosidase (GCD), is currently treated by enzyme replacement therapy using recombinant GCD (Cerezyme®) expressed in Chinese hamster ovary (CHO) cells. As complex glycans in mammalian cells d...