Liver-specific nucleic acid regulatory elements and methods and use thereof

摘要

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.

主权项

1. A nucleic acid expression cassette comprising an isolated nucleic acid regulatory element of 150 nucleotides or less for enhancing liver-specific gene expression, wherein the nucleic acid regulatory element comprises a polynucleotide selected from the group consisting of: SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 4, SEQ ID NO: 8, SEQ ID NO: 9, SEQ ID NO: 10, SEQ ID NO: 11, and a polynucleotide having 95% identity to any of these sequences, wherein the nucleic acid regulatory element is operably linked to a promoter and a heterologous transgene.