Enzyme replacement for lysosomal diseases.

Abstract:

:Following the demonstration of the nature of the enzymatic defects in the sphingolipid storage disorders in the mid-1960s, consideration was directed to the development of therapy for patients with these conditions. High on the list of possibilities was enzyme supplementation or replacement. Many years of arduous investigation and the development of novel protein targeting strategies were required to bring this concept to fruition. Enzyme replacement therapy (ERT) was eventually shown to be extraordinarily effective for patients with Gaucher disease, the most prevalent metabolic storage disorder of humans. Demonstration of the benefit of ERT in this disorder led to the extension of this approach to the treatment of other lysosomal storage disorders. This review presents the current status and anticipated developments in this field.

journal_name

Annu Rev Med

authors

Brady RO

doi

10.1146/annurev.med.57.110104.115650

subject

Has Abstract

pub_date

2006-01-01 00:00:00

pages

283-96

eissn

0066-4219

issn

1545-326X

journal_volume

57

pub_type

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