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.
Patent No.:
Date of Patent:
Apr. 13, 2021
Filed:
Feb. 02, 2017
Applicant:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Inventors:
Christian Hinderer, Philadelphia, PA (US);
James M. Wilson, Philadelphia, PA (US);
Assignee:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); A61K 48/00 (2006.01); C12N 9/24 (2006.01); C12N 15/86 (2006.01); A61P 3/00 (2006.01); C12N 15/864 (2006.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); A61K 48/0075 (2013.01); A61P 3/00 (2018.01); C12N 9/2402 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); C12Y 302/01076 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/15 (2013.01);
Abstract
A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.