Antiprogestin-controllable transgene regulation in vivo.

Gene control systems that provide temporal and spatial regulation of transgene expression in response to orally delivered drugs are needed for advances in functional genomics, models of human disease and gene therapy. A regulation system based on the altered binding and activation properties of a truncated ligand-binding domain derived from the progesterone receptor has been shown to be effective in providing tissue-specific, antiprogestin-controllable gene expression in transgenic mice, transgenic fruit flies and animals that have been administered viral-based or plasmid-based gene therapy vectors.