Muscle-specific nucleic acid regulatory elements and methods and use thereof
AssigneesVrije Universiteit Brussel, Vib Vzw, Universiteit Gent
InventorsThierry Vandendriessche, Marinee Chuah, Pieter De Bleser
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Abstract
The present invention relates to nucleic acid regulatory elements that are able to enhance muscle-specific expression of genes, in particular 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 muscle-directed gene therapy, and for vaccination purposes.
Claims16
A nucleic acid expression cassette comprising at least one nucleic acid regulatory element for enhancing muscle-specific gene expression, operably linked to a promoter and a heterologous transgene, wherein said nucleic acid regulatory element comprises the sequence set forth in SEQ ID NO:1, or a functional fragment of the sequence set forth in SEQ ID NO:
- 1, wherein said functional fragment consists of a nucleotide sequence selected from any one of the following:
- positions 143 to 233 of SEQ ID NO:1, positions 240 to 310 of SEQ ID NO:
- 1, or positions 90 to 233 of SEQ ID NO:
- 1, or comprises at least 300 contiguous nucleotides from the sequence set forth in SEQ ID NO:1.
A vector comprising at least one nucleic acid regulatory element for enhancing muscle-specific gene expression, operably linked to a promoter and a transgene wherein said nucleic acid regulatory element comprises the sequence set forth in SEQ ID NO:1, or a functional fragment of the sequence set forth in SEQ ID NO:
- 1, wherein said functional fragment consists of a nucleotide sequence selected from any one of the following:
- positions 143 to 233 of SEQ ID NO:1, positions 240 to 310 of SEQ ID NO:1, or positions 90 to 233 of SEQ ID NO:1, or comprises at least 300 contiguous nucleotides from the sequence set forth in SEQ ID NO:1.
Dependencies are inferred from the claim text.