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:
Feb. 16, 2016

Filed:

Oct. 28, 2014
Applicant:

Research Development Foundation, Carson City, NV (US);

Inventors:

Didier Trono, Vufflens-le-Chateau, CH;

Romain N. Zufferey, Palo Alto, CA (US);

Assignee:

Research Development Foundation, Carson City, NV (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/86 (2006.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); C12N 7/00 (2013.01); A61K 48/00 (2013.01); C12N 2740/15043 (2013.01); C12N 2740/16043 (2013.01); C12N 2740/16052 (2013.01); C12N 2740/16061 (2013.01); C12N 2830/38 (2013.01); C12N 2830/48 (2013.01); C12N 2830/50 (2013.01); C12N 2840/203 (2013.01); C12N 2840/44 (2013.01);
Abstract

The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5' leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosafety and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.


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