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.
Claims23
A 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 trans-acting factor in the nucleus of a target cell,
- 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 trans-acting factor to said cis-acting element.
A 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, wherein the cis-acting element is a tetO-operator,
- 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 trans-acting factor in the nucleus of a target cell, wherein the DNA binding domain of the trans-acting factor is a Tet repressor,
- 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 trans-acting factor to said cis-acting element, wherein the domain capable of maintaining the trans-acting factor in the nucleus of a target cell is EBNA1.
Dependencies are inferred from the claim text.