关于将三种重组人α-半乳糖苷酶 A 药物（琼脂糖酶）纳入法布里小鼠培养成纤维细胞和器官/组织的比较研究

IF 1.8 4区医学 Q3 GENETICS & HEREDITY Molecular Genetics and Metabolism Reports Pub Date : 2024-07-15 DOI:10.1016/j.ymgmr.2024.101118

Takahiro Tsukimura, Tomoko Shiga, Tadayasu Togawa, Hitoshi Sakuraba

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引用次数: 0

摘要

使用重组人 α-半乳糖苷酶 A（α-Gal A）药物（激动苷酶）的酶替代疗法（ERT）已成功用于治疗法布里病，目前日本已有三种激动苷酶上市。为了比较这些药物的生化特性，特别是它们在培养成纤维细胞和法布里小鼠器官/组织中的结合情况，我们进行了体外、细胞和动物实验。

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Comparative study on incorporation of three recombinant human α-galactosidase A drugs (agalsidases) into cultured fibroblasts and organs/tissues of Fabry mice

Enzyme replacement therapy (ERT) with recombinant human α-galactosidase A (α-Gal A) drugs (agalsidases) has been successfully used for treatment of Fabry disease, and three kinds of agalsidases are now available in Japan. To compare the biochemical characteristics of these drugs, especially focusing on their incorporation into cultured fibroblasts and organs/tissues of Fabry mice, we performed in vitro, cell, and animal experiments.

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来源期刊

Molecular Genetics and Metabolism Reports Biochemistry, Genetics and Molecular Biology-Endocrinology

CiteScore

4.00

自引率

5.30%

发文量

105

审稿时长

33 days

期刊介绍： Molecular Genetics and Metabolism Reports is an open access journal that publishes molecular and metabolic reports describing investigations that use the tools of biochemistry and molecular biology for studies of normal and diseased states. In addition to original research articles, sequence reports, brief communication reports and letters to the editor are considered.