Enzyme replacement and enhancement therapies: Lessons from lysosomal disorders

被引：212

作者：

Desnick, RJ ^{[1
]}

Schuchman, EH ^{[1
]}

机构：

[1] NYU, Mt Sinai Sch Med, Dept Human Genet, New York, NY 10029 USA

来源：

NATURE REVIEWS GENETICS | 2002年 / 3卷 / 12期

基金：

美国国家卫生研究院;

关键词：

D O I：

10.1038/nrg963

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

The past decade has witnessed remarkable advances in our ability to treat inherited metabolic disorders, especially the lysosomal storage diseases, a group of more than 40 disorders, each of which is caused by the deficiency of a lysosomal enzyme or protein. During the past few years, both enzyme replacement and enhancement therapies have been developed to treat these disorders. This review discusses the successes and shortcomings of these therapeutic strategies, and the contributions that they have made to treating lysosomal storage diseases.

引用

页码：954 / 966

页数：13

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