Novel nucleic acid construct useful in gene therapy comprising an hormone responsive element and transgene in which the hormone responsive element is not functionally linked to the transgene

摘要

A nucleic acid construct (I) comprising an hormone responsive element (HRE) and a transgene (T), is new. Independent claims are also included for the following: (1) a nucleic acid construct (II), comprising at least one HRE and a (T), where one of the HREs is not functionally linked to the transgene; (2) a vector (III) comprising (I), or (II); (3) a transformed cell or transgenic organism comprising (I), (II) or (III); (4) a composition of matter (IV) comprising (II), and/or (III), and in which HRE is coupled to a hormone-hormone receptor complex; (5) preparing (IV) involves admixing (II) with the hormone receptor and the hormone; (6) a pharmaceutical composition comprising (II), (III) and/or (IV); (7) gene transfer comprising administering a gene transfer agent (GTA) comprising (I), or (IV) to an organism or to a cellular system; (8) delivering (D) a nucleic acid construct encoding (T) to be expressed in the cells of the organism or the cells of the cellular system, into an organism or into a cellular system involves administering GTA comprising (I), or (IV) to the organism or cellular system so that the hormone in the composition interacts with the cell membrane and enhances diffusion and transport of the nucleic acid that is coupled to the hormone-hormone receptor complex; (9) use of a steroid hormone for preparing GTA; and (10) gene transfer comprising administering (I) to a organism or a cellular system in which (I) contains a transgene and is encapsulated in a steroid hormone.