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DRUG FOR THE TREATMENT OF CHOLESTEROL ACCUMULATION DISORDERS, AND SCREENING METHOD FOR SAME

外国特許コード F150008566
整理番号 (S2014-0214-N0)
掲載日 2015年11月26日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2014JP081969
国際公開番号 WO 2015083736
国際出願日 平成26年12月3日(2014.12.3)
国際公開日 平成27年6月11日(2015.6.11)
優先権データ
  • 特願2013-252174 (2013.12.5) JP
  • 特願2014-135865 (2014.7.1) JP
発明の名称 (英語) DRUG FOR THE TREATMENT OF CHOLESTEROL ACCUMULATION DISORDERS, AND SCREENING METHOD FOR SAME
発明の概要(英語) 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.
  • 出願人(英語)
  • ※2012年7月以前掲載分については米国以外のすべての指定国
  • NATIONAL UNIVERSITY CORPORATION KUMAMOTO UNIVERSITY
  • 発明者(英語)
  • ERA TAKUMI
  • IRIE TETSUMI
国際特許分類(IPC)
指定国 National States: AE AG AL AM AO AT AU AZ BA BB BG BH BN BR BW BY BZ CA CH CL CN CO CR CU CZ DE DK DM DO DZ EC EE EG ES FI GB GD GE GH GM GT HN HR HU ID IL IN IR IS JP KE KG KN KP KR KZ LA LC LK LR LS LU LY MA MD ME MG MK MN MW MX MY MZ NA NG NI NO NZ OM PA PE PG PH PL PT QA RO RS RU RW SA SC SD SE SG SK SL SM ST SV SY TH TJ TM TN TR TT TZ UA UG US UZ VC VN ZA ZM ZW
ARIPO: BW GH GM KE LR LS MW MZ NA RW SD SL SZ TZ UG ZM ZW
EAPO: AM AZ BY KG KZ RU TJ TM
EPO: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
OAPI: BF BJ CF CG CI CM GA GN GQ GW KM ML MR NE SN TD TG
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