Vectors conditionally expressing protein
AssigneeIntrexon Corporation
InventorsJeremiah F. Roeth, Charles C. Reed, Brandon Cuthbertson, Sunil Chada, William E. Fogler, Fayaz Khazi
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Abstract
This invention relates to the field of therapeutics. Disclosed are methods of conditionally expressing erythropoietin under the control of an ecdysone receptor-based gene expression modulation system in the presence of activating ligand and uses for therapeutic purposes in animals. The methods of the invention cause an in vivo increase in the expression of erythropoietin and an increase in the hematocrit or volume percentage of red blood cells in blood after administration of the ligand.
Claims28
A method of inducing, regulating, or enhancing erythropoietin (EPO) expression in a mammal, wherein the method comprises
- (a) administering an adeno-associated virus to the mammal, wherein the virus comprises a polynucleotide encoding EPO;
- and
- (b) administering an activator ligand which induces EPO expression from the virus polynucleotide encoding EPO,
- wherein the adeno-associated virus is administered intramuscularly,
- wherein the adeno-associated virus further comprises an ecdysone receptor (EcR)-based gene switch, wherein the gene switch comprises at least one transcription factor sequence operably linked to a first promoter, wherein at least one transcription factor encoded by the at least one transcription factor sequence is a ligand-dependent transcription factor,
- wherein the adeno-associated virus further comprises a second promoter operably linked to the polynucleotide encoding EPO, wherein the second promoter is activated by the at least one ligand-dependent transcription factor following administration of activator ligand,
- wherein EPO expression is increased in the mammal, and
- wherein the hematocrit or volume percentage of red blood cells in blood is increased in the mammal by at least 25%, compared with the hematocrit or volume percentage of red blood cells prior to administration of ligand.
Dependencies are inferred from the claim text.