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.

Abstract: The present invention relates to nucleic acid regulatory elements that are able to enhance heart-and/or muscle-targeted expression of genes, in particular heart- and muscle-targeted gene expression, more particularly gene expression in cardiomyocytes, 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 heart- and/or muscle-directed gene therapy, e.g. for the treatment of cardiovascular diseases and disorders and muscle disorders, as well as other diseases and disorders that may benefit from high transgene expression in heart and/or muscle cells or tissue, and for vaccination purposes.

Abstract: Nucleic acid regulatory elements that are able to enhance muscle-specific expression of genes, in particular expression in muscle cells and/or tissues such as in diaphragm, smooth muscle, heart and/or skeletal muscle, including at least two diaphragm-specific regulatory elements and a heart- and skeletal muscle-specific regulatory element. Expression cassettes and vectors containing these nucleic acid regulatory elements, as well as uses thereof such as applications using gene therapy of muscle-related disorders, more particularly diaphragm, heart and/or skeletal muscle-directed gene therapy, and for vaccination purposes.

Abstract: Nucleic acid regulatory elements that are able to enhance muscle-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 present invention is particularly useful for applications using gene therapy, more particularly muscle-directed gene therapy.

Abstract: The present invention relates to nucleic acid regulatory elements that are able to enhance liver-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 present invention is particularly useful for applications using gene therapy, more particularly liver-directed gene therapy, and for vaccination purposes.

Abstract: The present invention relates to a nucleic acid molecule encoding human albumin for increasing the levels and/or activity of a protein or polypeptide encoded by a transgene, comprising a sequence defined by SEQ ID NO: 14 or a sequence having at least 80% sequence identity to said sequence, its use in nucleic acid expression cassettes and vectors containing liver-specific regulatory elements and codon-optimized factor IX, factor VIII, factor VII or factor VIIa transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A, hemophilia B or factor VII deficiency.

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.

Abstract: The present invention relates to nucleic acid regulatory elements that are able to enhance cardiac-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 present invention is particularly useful for applications using gene therapy.

Abstract: The present invention relates to nucleic acid regulatory elements that are able to enhance cardiac-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 present invention is particularly useful for applications using gene therapy.