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US 2017/0304464 A1Published application
Regulatable adeno-associated virus (aav) vector
OwnerSebastian KÜGLERinventor
Life of this patent
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Abstract
The present invention relates to regulatable adeno-associated virus (AAV) vectors as well as to their use in gene therapy. It further relates to corresponding nucleic acid molecules, host cells, non-human transgenic animals, pharmaceutical compositions and kits.
Claims25
An adeno-associated virus (AAV) vector comprising
- (i) a first expression cassette directing the expression of a regulator protein under the control of a first promoter, wherein the regulator protein is activated in the presence of an activator molecule, and
- (ii) a second expression cassette directing the expression of a molecule of interest, wherein the second expression cassette comprises a promoter region, and the expression of the molecule of interest is induced by binding of the activated regulator protein to the promoter region,
- wherein the first expression cassette and the second expression cassette are arranged in a tail-to-head configuration.
An adeno-associated virus (AAV) vector construct comprising
- (i) a first expression cassette directing the expression of a regulator protein under the control of a first promoter, wherein the regulator protein is activated in the presence of an activator molecule, and
- (ii) a second expression cassette comprising a multiple cloning site allowing the insertion of a coding sequence for a molecule of interest, wherein the second expression cassette comprises a promoter region, and the expression of the molecule of interest is induced by binding of the activated regulator protein to the promoter region,
- wherein the first expression cassette and the second expression cassette are arranged in a tail-to-head configuration.
Dependencies are inferred from the claim text.