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.

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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

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Updated date 2019/6/13
Information
(External link)
https://www.j-platpat.inpit.go.jp/c1800/PU/WO-A-2018-230505/AC5F242BAC1F92ADC2A10DF3F5472FFB425222A779D7C8833D06990BE45EE768/50/en