Lysosomal storage diseases: pathological mechanisms and therapeutic options

被引：1
|
作者：
Futerman, AH [1 ]
机构：
[1] Weizmann Inst Sci, Dept Biol Chem, IL-76100 Rehovot, Israel
来源：
M S-MEDECINE SCIENCES | 2005年 / 21卷
关键词：
D O I：
10.1051/medsci/20052111s16
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
More than 40 lysosomal disorders are known and classified into broad categories based on the nature of the undegraded metabolites. In this review, the defective enzymatic activities are analyzed, and the pathology of the disorders are described. Various treatments are discussed, including enzyme replacement therapy, which is the most successful treatment available today.
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收藏
页码：16 / 19
页数：4
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