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US 10,471,157 B2Granted
Liver-specific nucleic acid regulatory elements and methods and use thereof
AssigneesVib Vzw, Universiteit Gent
InventorsMarinee Chuah, Thierry Vandendriessche, Pieter De Bleser
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Abstract
Described are 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. These are particularly useful for applications using gene therapy.
Claims20
A nucleic acid expression cassette comprising:
- a nucleic acid regulatory element of 100 nucleotides or less that enhances liver-specific gene expression,
- wherein the nucleic acid regulatory element comprises SEQ ID NO:7, or a polynucleotide having at least 95% identity to SEQ ID NO:7, and
- wherein the nucleic acid regulatory element is operably linked to a promoter and a heterologous transgene.
A nucleic acid expression cassette comprising:
- at least two nucleic acid regulatory elements that enhance liver-specific gene expression, each of the at least two nucleic acid regulatory elements comprising:
- a first polynucleotide of 100 nucleotides or less comprising SEQ ID NO:7 or a polynucleotide having at least 95% identity to SEQ ID NO:7;
- and
- a second nucleic acid regulatory element of 90 nucleotides or less comprising SEQ ID NO:3 or a polynucleotide having at least 95% identity to SEQ ID NO:3.
1 more element
Dependencies are inferred from the claim text.