Abstract |
Enzyme therapies for lysosomal storage diseases have developed over the past decade into the standard-of-care for affected patients. Such therapy for Gaucher disease has been the prototype, using natural source or recombinant forms of human acid beta-glucosidase (GCase). In Gaucher disease, macrophages are the repository for the pathological lipid and the target for delivery of GCase. The macrophage mannose receptor provides a Trojan horse for intracellular delivery of intravenously administered GCase (man-GCase) with mannosyl-terminated oligosaccharide chains. Passage through several hostile compartments (e.g., plasma) leads to inefficient delivery of man-GCase to macrophage lysosomes. However, regular infusions of man-GCase re-establishes health in affected patients. Similar results are being obtained in several other lysosomal storage diseases. Evolving gene and chaperone approaches provide alternative treatment strategies.
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Authors | Gregory A Grabowski |
Journal | Expert opinion on drug delivery
(Expert Opin Drug Deliv)
Vol. 3
Issue 6
Pg. 771-82
(Nov 2006)
ISSN: 1742-5247 [Print] England |
PMID | 17076599
(Publication Type: Journal Article, Review)
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Chemical References |
- Recombinant Proteins
- Glucosylceramidase
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Topics |
- Animals
- Drug Delivery Systems
(methods)
- Gaucher Disease
(drug therapy, enzymology)
- Glucosylceramidase
(administration & dosage, chemistry, therapeutic use)
- Humans
- Lysosomal Storage Diseases
(drug therapy, enzymology)
- Recombinant Proteins
(administration & dosage, chemistry, therapeutic use)
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