Endothelium-specific nucleic acid regulatory elements and methods and use thereof
AssigneeVrije Universiteit Brussel
InventorsMarinee Chuah, Thierry Vandendriessche
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Abstract
The disclosure provides nucleic acid regulatory elements that are able to enhance endothelial cell-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The nucleic acid regulatory elements, methods of employing these regulatory elements, uses of these elements, and expression cassettes and vectors containing these nucleic acid regulatory elements are particularly useful for applications using gene therapy, more particularly endothelial cell-directed gene therapy, and for vaccination purposes.
Claims15
A method for treating or preventing an endothelial cell-related disease or disorder, the method comprising:
- i) administering a therapeutically effective amount of a nucleic acid expression cassette;
- a vector comprising said nucleic acid expression cassette;
- or a pharmaceutical composition comprising said nucleic acid expression cassette or said vector, and a pharmaceutically acceptable carrier, to a subject in need thereof,
- wherein said nucleic acid expression cassette comprises one or more nucleic acid regulatory element for enhancing endothelial cell-specific gene expression comprising the sequence of SEQ ID NO:
- 22, operably linked to a promoter.
A method for enhancing transgene expression in endothelial cells in a subject undergoing endothelial cell-directed gene therapy, the method comprising:
- i) administering to the subject a nucleic acid expression cassette;
- a vector comprising said nucleic acid expression cassette;
- or a pharmaceutical composition comprising said nucleic acid expression cassette or said vector, and a pharmaceutically acceptable carrier, wherein said nucleic acid expression cassette comprises one or more nucleic acid regulatory element for enhancing endothelial cell-specific gene expression comprising the sequence of SEQ ID NO:
- 22, operably linked to a promoter and a transgene;
- and
- ii) expressing a therapeutically effective amount of the transgene product in endothelial cells of the subject.
A method for vaccinating a subject, the method comprising:
- i) administering to the subject a nucleic acid expression cassette;
- a vector comprising said nucleic acid expression cassette;
- or a pharmaceutical composition comprising said nucleic acid expression cassette or said vector, and a pharmaceutically acceptable carrier, wherein said nucleic acid expression cassette comprises one or more nucleic acid regulatory element for enhancing endothelial cell-specific gene expression comprising the sequence of SEQ ID NO:
- 22, operably linked to a promoter and a transgene;
- and
- ii) expressing an immunologically effective amount of the transgene product in endothelial cells of the subject.
Dependencies are inferred from the claim text.