Toward enzyme therapy for lysosomal storage disease

R. J. Desnick, S. R. Thorpe, M. B. Fiddler

Research output: Contribution to journalReview articlepeer-review

93 Scopus citations

Abstract

The lysosomal storage diseases have become models of the progress in the delineation of the molecular pathology of inherited metabolic disorders. It is, indeed, a fact that the recent advances in the characterization of the molecular biology of the lysosomal apparatus and its enzymatic defects have laid the foundation for the development of therapeutic endeavors for these disorders. The prospects for enzyme therapy will depend on the ingenuity to develop molecular and cellular strategies necessary to normalize the metabolism in these and possibly other metabolic disorders. This review (with 181 references) focusses on the past and recent achievements, as well as the current barriers encountered on the pathway toward enzyme therapy for the lysosomal storage diseases.

Original languageEnglish
Pages (from-to)57-99
Number of pages43
JournalPhysiological Reviews
Volume56
Issue number1
DOIs
StatePublished - 1976
Externally publishedYes

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