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AssigneeNational University Corporation Kumamoto University
InventorsTakumi Era, Tetsumi Irie
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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.
A method for treating a lysosomal disease in a subject, comprising administrating to said subject in need thereof a therapeutically effective amount of hydroxypropyl-γ-cyclodextrin as an active ingredient.
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