Invention Grant
- Patent Title: Therapeutic agent for diseases associated with abnormalities in dystroglycan sugar chain modification and method for assaying associated enzyme
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Application No.: US16324194Application Date: 2017-08-18
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Publication No.: US11040087B2Publication Date: 2021-06-22
- Inventor: Tatsushi Toda , Kazuhiro Kobayashi , Motoi Kanagawa , Tamao Endo , Hiroshi Manya , Yoshinao Wada , Michiko Tajiri
- Applicant: NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY , TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY , OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- Applicant Address: JP Hyogo; JP Tokyo; JP Osaka
- Assignee: NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY,TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY,OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- Current Assignee: NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY,TOKYO METROPOLITAN GERIATRIC HOSPITAL AND INSTITUTE OF GERONTOLOGY,OSAKA PREFECTURAL HOSPITAL ORGANIZATION
- Current Assignee Address: JP Hyogo; JP Tokyo; JP Osaka
- Agency: Kilyk & Bowersox, P.L.L.C.
- Priority: JPJP2016-160390 20160818
- International Application: PCT/JP2017/029600 WO 20170818
- International Announcement: WO2018/034334 WO 20180222
- Main IPC: A61K38/45
- IPC: A61K38/45 ; A61P21/00 ; G01N33/68
Abstract:
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.
Information query
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