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Delivery of lysosomal enzymes for therapeutic use: glucocerebrosidase as an example.

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.
AuthorsGregory A Grabowski
JournalExpert opinion on drug delivery (Expert Opin Drug Deliv) Vol. 3 Issue 6 Pg. 771-82 (Nov 2006) ISSN: 1742-5247 [Print] England
PMID17076599 (Publication Type: Journal Article, Review)
Chemical References
  • Recombinant Proteins
  • Glucosylceramidase
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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