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. |