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:
Sep. 19, 2017

Filed:

Apr. 07, 2015
Applicants:

Ucl Business Plc, London, GB;

Thrombosis Research Institute, Chelsea, GB;

St. Jude Children's Research Hospital, Memphis, TN (US);

Inventors:

Amit Nathwani, London, GB;

Natalie Ward, London, GB;

Adrian Thrasher, London, GB;

Edward Tuddenham, London, GB;

John Mcvey, London, GB;

John Gray, Memphis, TN (US);

Andrew Davidoff, Memphis, TN (US);

Assignees:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C12N 15/63 (2006.01); A61K 48/00 (2006.01); C07K 14/755 (2006.01); C12N 15/86 (2006.01); A61K 38/37 (2006.01); C12N 7/00 (2006.01); A01K 67/00 (2006.01); A61K 38/00 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0058 (2013.01); A61K 38/37 (2013.01); C07K 14/755 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); C12N 2710/10041 (2013.01); C12N 2710/14143 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01); C12N 2830/008 (2013.01);
Abstract

An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.


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