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US 2007/0179113 A1Published application
GM-CSF gene therapy for Crohn's disease using an improved regulated expression system
AssigneeSchering Aktiengesellachaft
InventorsMaxine Bauzon, Richard Harkins, Terry Hermiston, Peter Kretschmer, Harald Petry, Paul Szymanski
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Abstract
The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.
Claims78
A regulated gene expression system comprising a vector comprising:
- a) a first gene expression cassette comprising
- i) a first nucleic acid sequence encoding a GM-CSF;
- and
- ii) a first promoter and first polyadenylation (polyA) site operably linked to said first nucleic acid sequence;
- wherein the expression of said first nucleic acid sequence and/or the activity of the expressed polypeptide is regulated in the presence of a regulator molecule (RM);
- and
- b) a second gene expression cassette comprising
- i) a second nucleic acid sequence encoding said RM;
- and
- ii) a second promoter and a second polyA site operably linked to said second nucleic acid sequence;
- wherein said RM is expressed and regulates the expression and/or activity of said first nucleic acid sequence.
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Dependencies are inferred from the claim text.