NATIONAL UNIVERSITY CORPORATION KUMAMOTO UNIVERSITY;NIHON SHOKUHIN KAKO CO., LTD.
发明人:
Takumi ERA,Tetsumi IRIE
申请号:
US15101109
公开号:
US20170216342A1
申请日:
2014.12.03
申请国别(地区):
US
年份:
2017
代理人:
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.