Enzyme replacement therapy for Gaucher disease

Joel Charrow*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

27 Scopus citations


Gaucher disease is the most common lysosomal storage disease, and the first lysosomal storage disease for which a specific therapy has been developed. Enzyme replacement therapy, with glucocerebrosidase purified from human placentae, was introduced in 1991. Recombinant human glucocerebrosidase, produced by Chinese hamster ovary cells in tissue culture, became available in 1994 and has replaced the placenta-derived product. These therapies have revolutionized the care of patients with type 1 Gaucher disease, reversing many of the pathological consequences of this disease, and preventing further progression. Furthermore, they have served as a model for the treatment of other lysosomal storage diseases and inborn errors of metabolism.

Original languageEnglish (US)
Pages (from-to)121-131
Number of pages11
JournalExpert Opinion on Biological Therapy
Issue number1
StatePublished - Jan 2009


  • Alglucerase
  • Enzyme replacement therapy
  • Gaucher disease
  • Glucocerebrosidase
  • Imiglucerase
  • Inborn errors of metabolism
  • Lipidosis
  • Sphingolipidosis

ASJC Scopus subject areas

  • Drug Discovery
  • Clinical Biochemistry
  • Pharmacology


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