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Synonyms: Elelyso® | PRGCD
taliglucerase alfa is an approved drug (FDA (2012))
Compound class:
Peptide or derivative
Comment: Taliglucerase alfa is the first approved recombinant drug to be produced in genetically engineered plant cells. Carrot cells have been genetically modified to express human β-glucocerebrosidase. The fully active enzyme is then extracted from the cultured cells.
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| No information available. |
Summary of Clinical Use  |
| Taliglucerase alfa is approved to be used as an enzyme replacement therapy for Gaucher's disease patients, whose own β-glucocerebrosidase is non-functional. |
| Mechanism Of Action and Pharmacodynamic Effects |
| Taliglucerase alfa is a glucocerebroside hydrolytic enzyme. The enzyme β-glucocerebrosidase (GBA, P04062), is a lysosomal enzyme that hydrolyses glucocerebroside as a step in glycolipid metabolism. β-glucocerebrosidase activity is impaired in Gaucher's disease and this results in accumulation of glucocerebroside. |
