- 专利标题： Therapeutic agent for diseases associated with abnormalities in dystroglycan sugar chain modification and method for assaying associated enzyme
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申请号： US16324194申请日： 2017-08-18
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公开(公告)号： US11040087B2公开(公告)日： 2021-06-22
- 发明人: Tatsushi Toda , Kazuhiro Kobayashi , Motoi Kanagawa , Tamao Endo , Hiroshi Manya , Yoshinao Wada , Michiko Tajiri
- 申请人： NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY , TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY , OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- 申请人地址： JP Hyogo; JP Tokyo; JP Osaka
- 专利权人： NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY,TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY,OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- 当前专利权人： NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY,TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY,OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- 当前专利权人地址： JP Hyogo; JP Tokyo; JP Osaka
- 代理机构： Kilyk & Bowersox, P.L.L.C.
- 优先权： JPJP2016-160390 20160818
- 国际申请： PCT/JP2017/029600 WO 20170818
- 国际公布： WO2018/034334 WO 20180222
- 主分类号： A61K38/45
- IPC分类号： A61K38/45 ; A61P21/00 ; G01N33/68
摘要：
Provided is a therapeutic agent that effectively acts on a disease associated with abnormal glycosylation of dystroglycan. Also provided is a testing method for diseases associated with abnormal glycosylation of dystroglycan. Specifically, provided is a therapeutic agent for diseases associated with abnormal glycosylation of dystroglycan, containing CDP-ribitol as an active ingredient. Ribitol-phosphate is important in the glycan structure of dystroglycan. In order for ribitol-phosphate to be incorporated into a dystroglycan glycan, a material therefor (sugar donor) is required. In the present invention, it has been found for the first time that CDP-ribitol serves as the sugar donor. It has been confirmed that the glycan of ISPD-deficient cells can be restored by administering CDP-ribitol. Thus, the present invention, which allows CDP-ribitol to be utilized for supplementation therapy, has been completed.
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