药物伴侣疗法作为治疗戈谢病的一种策略。
Pharmacologic chaperoning as a strategy to treat Gaucher disease.
作者信息
Yu Zhanqian, Sawkar Anu R, Kelly Jeffery W
机构信息
Department of Chemistry and The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA 92037, USA.
出版信息
FEBS J. 2007 Oct;274(19):4944-50. doi: 10.1111/j.1742-4658.2007.06042.x.
Abstract
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.
摘要
我们简要介绍最常见的溶酶体贮积症——戈谢病，简要描述美国食品药品监督管理局批准的改善戈谢病的策略，然后概述新兴的药物伴侣策略，该策略有望治愈这种疾病。
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