Patent Portfolio
Method for screening drugs for treatment of X-linked sideroblastic anemia
| Ref. No. | AJ186 |
|---|---|
| Appl. No. （filing date） |
PCT/JP2018/022245（2018/6/11） |
| Pub. No. （Pub. date） |
WO2018/230505（2018/12/20） |
| Priority claim No. （Priority date） |
JP2017-115232 (2017/06/12) |
| Patent No. | - |
| Inventor | YOSHIDA, Yoshinori, etc. |
| Assignee | Kyoto University |
| Title of Invention | Method for screening drugs for treatment of X-linked sideroblastic anemia |
| Abstract | The present invention provides a method for screening drugs for treatment of X-linked sideroblastic anemia (XLSA) comprising a step of differentiating stem cells (incl. iPS cells) derived from an XLSA patient, in which an X chromosome without an ALAS2 gene mutation is deactivated and an X chromosome having an ALAS2 gene mutation is activated, into red blood cells in the presence and absence of a test substance, and measuring the differentiation efficiency. |
| Comments |
- |
| Updated date | 2019/6/13 |
| Information (External link) |
https://www.j-platpat.inpit.go.jp/c1800/PU/WO-A-2018-230505/AC5F242BAC1F92ADC2A10DF3F5472FFB425222A779D7C8833D06990BE45EE768/50/en |