The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 17, 2020

Filed:

Dec. 03, 2014
Applicants:

National University Corporation Kumamoto University, Kumamoto, JP;

Nihon Shokuhin Kako Co., Ltd., Tokyo, JP;

Inventors:

Takumi Era, Kumamoto, JP;

Tetsumi Irie, Kumamoto, JP;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 31/74 (2006.01); A61K 9/00 (2006.01); G01N 33/92 (2006.01); G01N 33/50 (2006.01); A61K 31/724 (2006.01); C12N 5/074 (2010.01);
U.S. Cl.
CPC ...
A61K 31/724 (2013.01); A61K 9/0019 (2013.01); C12N 5/0696 (2013.01); G01N 33/5073 (2013.01); G01N 33/92 (2013.01); G01N 2500/04 (2013.01); G01N 2500/10 (2013.01); G01N 2800/04 (2013.01);
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.


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