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filingDate 2018-06-11-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b8c9e6a301de0769f81307ae05917b4a
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publicationDate 2018-12-20-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2018230505-A1
titleOfInvention Method for screening drugs for treatment of x-linked sideroblastic anemia
abstract The present invention relates to a method for screening drugs for the treatment of X-linked sideroblastic anemia that includes the following steps: (a) differentiating stem cells in which an X chromosome without an ALAS2 gene mutation from an X-linked sideroblastic anemia patient (XLSA) 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; and (b) assessing that the test substance has the possibility of being a drug for the treatment of X-linked sideroblastic anemia when the differentiation efficiency in the presence of the test substance is higher than the differentiation efficiency in the absence of the test substance.
isCitedBy http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022145144-A1
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