Hormone-hormone receptor complexes and nucleic acid constructs and their use in gene therapy

摘要

The invention relates to the use of a nucleic acid contruct comprising at least one hormone responsive element and a transgene for preparing an agent for gene transfer. It further relates to particular nucleic acid contructs comprising at least one hormone responsive element and a transgene, wherein one of said at least one hormone responsive elements is not functionally linked to the transgene, vectors comprising such nucleic acid contructs and compositions of matter comprising such nucleic acid constructs wherein the hormone responsive elements of the constructs are coupled to a hormone-hormone receptor complex. The nucleic acid constructs, plasmids, and compositions of matter of the invention have applications in gene therapy, particularly in the treatment of human blood clotting disorders, such as hemophilia. They may also be used to up- or down-regulate target genes and for the delivery of vaccines.