Differences in cleavage of globotriaosylceramide and its derivatives accumulated in organs of young Fabry mice following enzyme replacement therapy

详细信息    查看全文

• 作者：Takashi Kodama^a ; Takahiro Tsukimura^a ; Ikuo Kawashima^b ; Atsuko Sato^c ; Hitoshi Sakuraba^c ; Tadayasu Togawa^a ; ^{tadayasu@my-pharm.ac.jp}
• 关键词：Fabry disease ; α-Galactosidase A ; α-Galactosidase A knockout mouse ; Globotriaosylceramide ; Globotriaosylsphingosine ; Enzyme replacement therapy
• 刊名：Molecular Genetics and Metabolism
• 出版年：2017
• 出版时间：January-February 2017
• 年：2017
• 卷：120
• 期：1-2
• 页码：116-120
• 全文大小：290 K
• 卷排序：120

文摘

The contents of Gb3 and lyso-Gb3 in the tissues measured in young Fabry mice Recurrent administration of agalsidase beta had prominent effects in terms of degradation of Gb3. Some Gb3 isoforms accumulated in the kidneys largely escaped from degradation.