Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
AssigneeVrije Universiteit Brussel
InventorsLay Khim Chuah, Thierry Vandendriessche, Warut Tulalamba
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Abstract
The present invention relates to nucleic acid regulatory elements that are able to enhance diaphragm-specific expression of genes, in particular expression in diaphragm as such, or in combination with expression in cardiac muscle and/or skeletal muscle, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly diaphragm-directed gene therapy, and for vaccination purposes.
Claims19
A method for enhancing gene expression in diaphragm, skeletal muscle and heart tissue, comprising administering to a subject in need thereof, a nucleic acid regulatory element having a maximal length of 1000 nucleotides and comprising the sequence defined by SEQ ID NO:
- 2, a complement of the sequence, or a sequence hybridizing under stringent conditions to the nucleic acid regulatory element.
A nucleic acid expression cassette comprising at least one nucleic acid regulatory element having a maximal length of 1000 nucleotides, and comprising an isolated fragment of a naturally occurring alpha-actin-1 (ACTA-1) promoter sequence wherein said fragment is defined by the sequence SEQ ID NO:
- 2, a complement of the sequence, or a sequence hybridizing under stringent conditions to the nucleic acid regulatory element, operably linked to a promoter other than an ACTA-1 promoter, wherein the promoter is at least one of a diaphragm-specific promoter, a heart-specific promoter and a skeletal muscle-specific promoter.
Dependencies are inferred from the claim text.