Abstract
Following the discovery that insufficient activity of glucocerebrosidase was the metabolic defect in Gaucher disease, attention was directed to the possibility that the health of patients might be improved by the administration of purified exogenous enzyme. Glucocerebrosidase for this investigation was originally obtained from human placental tissue. Intravenous administration of this enzyme to patients with Gaucher disease reduced the amount of accumulated glucocerebroside in the liver and circulation. Enzyme subsequently obtained by a large-scale purification procedure required modification of its glycoform for the successful treatment of patients with Gaucher disease. The steps involved in this endeavor and benefit of appropriately modified glucocerebrosidase for patients with Gaucher disease are chronicled.
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Brady, R.O. (2015). Targeting Glucocerebrosidase to Macrophages for Effective Treatment of Patients with Gaucher Disease: Setting the Paradigm of a “Fit for Purpose” Approach to Enzyme Replacement Therapy. In: Rosenberg, A., Demeule, B. (eds) Biobetters. AAPS Advances in the Pharmaceutical Sciences Series, vol 19. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-2543-8_1
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