Abstract
Gene therapy is a promising route for the delivery of secreted therapeutic proteins but pharmacologic control of gene expression will generally be required for optimal safety and efficacy. We have developed a regulation system in which expression of a target gene is made dependent on a transcription factor (TF) that is activated by rapamycin or AP22594, a derivative with substantially reduced immunosuppressive activity. To minimize the potential for immunogenicity in a clinical setting the system has been built exclusively from human proteins.
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