Related Patent Info

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

Inventor ERA Takumi etc.
Assignee NATIONAL UNIVERSITY CORPORATION KUMAMOTO UNIVERSITY
Title of Invention DRUG FOR THE TREATMENT OF CHOLESTEROL ACCUMULATION DISORDERS, AND SCREENING METHOD FOR SAME
Abstract

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.