Preparation and application of mouse model for the study on chaperone therapy for Fabry disease

法布里病伴侣治疗研究小鼠模型的制备及应用

• 批准号: 20590300
• 负责人: ISHII Satoshi
• 金额: $ 3.08万
• 依托单位: Obihiro University of Agriculture and Veterinary Medicine
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Scientific Research (C)
• 财政年份: 2008
• 资助国家: 日本
• 起止时间: 2008 至 2010
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-20590300/
• 关键词: ファブリー病; シャペロン療法; 応用動物

The purpose of our study is to establish a mouse model for the chaperone therapy for Fabry disease. Fabry disease is an inherited disease caused by the deficient activity of α-galactosidase A and resulted to accumulation of glycosphingolipid, predominantly globotriaosylceramide (Gb3). The mouse model established previously was good for the determination of enzyme activity but not for the accumulation of Gb3 on the effect of drug candidates. In our present study, we prepared the transgenic mouse expressing human Gb3 synthase gene, and we succeeded a mouse model in which we can determine the effect of compounds on both enzyme activity and Gb3 content.

本研究的目的是建立Fabry病分子伴侣治疗的小鼠模型。法布里病是一种遗传性疾病，由α-半乳糖苷酶A活性缺陷引起，并导致鞘糖脂，主要是神经酰胺三己糖苷（Gb 3）的积累。先前建立的小鼠模型对于酶活性的测定是良好的，但对于Gb 3对候选药物的作用的积累不是很好。在本研究中，我们制备了表达人Gb 3合酶基因的转基因小鼠，并且我们成功地建立了小鼠模型，其中我们可以确定化合物对酶活性和Gb 3含量的影响。

项目成果

Pharmacological chaperone therapy for Fabry disease.

• DOI: 10.2183/pjab.88.18
• 发表时间: 2012
• 期刊: Proceedings of the Japan Academy. Series B, Physical and biological sciences
• 作者: Ishii S

Increased globotriaosylceramide levels in a transgenic mouse expressing human α1,4-galactosyltransferase and a mouse model for Fabry disease

表达人 α1,4-半乳糖基转移酶的转基因小鼠和法布里病小鼠模型中三酰基神经酰胺水平升高

• 发表时间: 2011
• 期刊: Journal of Biochemistry
• 影响因子: 2.7
• 作者: Shiozuka C.;et al.
• 通讯作者: et al.

Fabry Disease, Chapter 29 : Pharmacological chaperone therapy for Fabry disease

法布里病，第 29 章：法布里病的药理学伴侣疗法

• 发表时间: 2010
• 作者: Fan J.Q.;Ishii S.
• 通讯作者: Ishii S.

Preclinical Efficacy and Safety of 1-Deoxygalactonojirimycin in Mice for Fabry Disease

• DOI: 10.1124/jpet.108.149054
• 发表时间: 2009-03-01
• 期刊: JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS
• 影响因子: 3.5
• 作者: Ishii, Satoshi;Chang, Hui-Hwa;Fan, Jian-Qiang
• 通讯作者: Fan, Jian-Qiang

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