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:
Nov. 27, 2018
Filed:
Mar. 13, 2014
Applicant:
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Inventors:
James M. Wilson, Philadelphia, PA (US);
Brittney L. Gurda, Collegeville, PA (US);
Assignee:
The Trustee of the University of Pennsylvania, Philadelphia, PA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 38/47 (2006.01); A61K 35/76 (2015.01); C12N 9/24 (2006.01); A61K 48/00 (2006.01); A61K 9/00 (2006.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
A61K 38/47 (2013.01); A61K 9/0019 (2013.01); A61K 35/76 (2013.01); A61K 48/0058 (2013.01); C12N 7/00 (2013.01); C12N 9/2402 (2013.01); C12N 2750/14143 (2013.01); C12Y 302/01076 (2013.01);
Abstract
A vector having an expression cassette having a hIDUA gene having a sequence of SEQ ID NO: 1 or a sequence at least about 95% identical thereto which encodes a functional human alpha-L-iduronidase is provided. The vector may be production vector or a rAAV8. Also provided are compositions containing these vectors and methods of treating MPSI and the symptoms associated with Hurler, Hurle-Scheie and Scheie syndromes.