Conditional gene vectors regulated in cis
AssigneeHelmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH)
InventorsWolfgang Hammerschmidt, Sibille Humme, Dagmar Pich, Aloys Schepers
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Abstract
The present invention is directed to a conditional gene vector system and to a host cell, which has been transfected with such a vector system. The present invention is further directed to a combined preparation comprising the vector system of the invention and an interfering agent. Furthermore, a pharmaceutical composition and its use in the treatment of hemophilia, diabetes, rheumatoid arthritis, genetic immunodeficiency, and graft versus host disease is provided.
Claims21
Conditional gene vector system comprising the following elements:
- a) a vector carrying a cis-acting element and one or more coding and/or non-coding sequences, and
- b) a trans-acting factor having a DNA binding domain capable of binding to a region of the cis-acting element and a domain capable of maintaining the protein in the nucleus of a target cell,
- characterized in that
- wherein the region of the cis-acting element and the DNA binding domain of the trans-acting factor capable of binding to the region of the cis-acting element are so selected that the nuclear maintenance of said vector carrying the cis-acting element in a target cell may be regulated by adding an agent capable of interfering with the binding of said tranis-acting factor to said cis-acting element.
Dependencies are inferred from the claim text.