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:
Jan. 17, 2023

Filed:

Apr. 24, 2015
Applicant:

The Trustees of the University of Pennsylvania, Philadelphia, PA (US);

Inventors:

James M. Wilson, Philadelphia, PA (US);

Daniel J. Rader, Philadelphia, PA (US);

Suryanrayan Somanathan, Boston, MA (US);

Assignee:
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 7/00 (2006.01); C12N 15/86 (2006.01); A61P 3/06 (2006.01); C07K 14/705 (2006.01); A61K 48/00 (2006.01); A61K 38/17 (2006.01);
U.S. Cl.
CPC ...
C07K 14/705 (2013.01); A61K 38/177 (2013.01); A61K 48/005 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01);
Abstract

A recombinant vector having an expression cassette is provided which comprises a modified human low density lipoprotein receptor (hLDLR) gene is provided, wherein said hLDLR gene encodes a modified hLDLR comprising (a) one or more of the following amino acid substitutions: L318H, N295D, H306D, V307D, N309A, D310N, L318H, and/or L318D; or (b) an amino acid substitution of any of (a) in combination with one or more of the following amino acid substitutions: K796, K809R and/or C818A. Also provided are pharmaceutical compositions containing this vector and uses therefor in lowering cholesterol and/or treating familial hypercholesterolemia.


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