Lentiviral System
AssigneeExpression Therapeutics LLC
InventorsHarrison BROWN, Gabriela DENNING
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Abstract
We disclose a lentiviral vector, for use in research, clinical, industrial, and other suitable applications. The novel lentiviral vectors disclosed herein introduce numerous novel elements which increase the safety profile of the vector without reducing the efficacy of the system. The novel lentiviral vectors are useful as safe and highly efficient transduction vectors for any application using or benefitting from transduction. We also disclose methods and compositions are provided that allow for high-level expression of a factor VIII polypeptide.
Claims20
An expression vector comprising:
- a. a lentiviral packaging system comprising,
- i. a first plasmid comprising a structural gene selected from a GAG gene, a POL gene, or both GAG and POL genes operatively linked to a first promoter;
- ii. a second plasmid comprising an ENV gene encoded by SEQ ID NO:
- 19 operatively linked to a second promoter;
- iii. a third plasmid comprising a REV gene encoded by SEQ ID NO:
- 36 operatively linked to a third promoter;
- and
- b. a lentiviral transfer plasmid;
- comprising a transgene operatively linked to a fourth promoter;
- c. wherein each of the first promoter, the second promoter, the third promoter, and the fourth promoter are unique one from the other.
A pharmaceutical composition comprising an an expression vector comprising:
- a. a lentiviral packaging system comprising,
- i. a first plasmid comprising a structural gene selected from a GAG gene, a POL gene, or both GAG and POL genes operatively linked to a first promoter;
- ii. a second plasmid comprising an ENV gene encoded by SEQ ID NO:
- 19 operatively linked to a second promoter;
- iii. a third plasmid comprising a REV gene encoded by SEQ ID NO:
- 36 operatively linked to a third promoter;
- and
- b. a lentiviral transfer plasmid;
- comprising a transgene operatively linked to a fourth promoter;
- wherein each of the first promoter, the second promoter, the third promoter, and the fourth promoter are unique one from the other.
A method of inducing blood clotting comprising administering an effective amount of a pharmaceutical composition, the pharmaceutical composition comprising an an expression vector comprising:
- a. a lentiviral packaging system comprising,
- i. a first plasmid comprising a structural gene selected from a GAG gene, a POL gene, or both GAG and POL genes operatively linked to a first promoter;
- ii. a second plasmid comprising an ENV gene encoded by SEQ ID NO:
- 19 operatively linked to a second promoter;
- iii. a third plasmid comprising a REV gene encoded by SEQ ID NO:
- 36 operatively linked to a third promoter;
- and
- b. a lentiviral transfer plasmid;
- comprising a transgene operatively linked to a fourth promoter;
- wherein each of the first promoter, the second promoter, the third promoter, and the fourth promoter are unique one from the other
Dependencies are inferred from the claim text.