发明名称 |
DRUG FOR THE TREATMENT OF CHOLESTEROL ACCUMULATION DISORDERS, AND SCREENING METHOD FOR SAME |
摘要 |
Provided is a pharmaceutical composition for the treatment of disorders such as Niemann-Pick disease and GM1 gangliosidosis which are caused by the storage of cholesterol, such as lysosomal storage disease. Also provided is a method for screening for said pharmaceutical compositions that uses iPS cell strains that phenocopy phentotypes of these disorders. Provided is a pharmaceutical composition for the treatment and/or prevention of lysosomal storage disease, characterized by containing hydroxypropyl-γ-cyclodextrin as an active ingredient. Also provided are an iPS cell strain derived from patients suffering from intractable disorders and prepared using a new temperature-sensitive Sendai virus vector, and a screening method for pharmaceuticals using said iPS cell strain. |
申请公布号 |
WO2015083736(A1) |
申请公布日期 |
2015.06.11 |
申请号 |
WO2014JP81969 |
申请日期 |
2014.12.03 |
申请人 |
NATIONAL UNIVERSITY CORPORATION KUMAMOTO UNIVERSITY |
发明人 |
ERA TAKUMI;IRIE TETSUMI |
分类号 |
A61K31/724;A61P1/16;A61P19/00;A61P25/00;A61P25/28;A61P27/02;C12N5/07;C12Q1/02 |
主分类号 |
A61K31/724 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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