Pharmacologic chaperoning as a strategy to treat Gaucher disease

被引：80

作者：

Yu, Zhanqian ^{[1
]}

Sawkar, Anu R. ^{[1
]}

Kelly, Jeffery W. ^{[1
]}

机构：

[1] Scripps Res Inst, Skaggs Inst Chem Biol, Dept Chem, La Jolla, CA 92037 USA

来源：

FEBS JOURNAL | 2007年 / 274卷 / 19期

关键词：

endoplasmic reticulum-associated degradation; folding; Gaucher disease; neuropathic Gaucher disease; pharmacologic chaperone; traficking; type 2 Gaucher disease; type 3 Gaucher disease;

D O I：

10.1111/j.1742-4658.2007.06042.x

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.

引用

页码：4944 / 4950

页数：7

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