Regulated promoters

Abstract

Over the past decades gene therapy has emerged as a promising approach for treatment of a variety of diseases including monogenic diseases, cancer, neurodegenerative disorders, autoimmune and inflammatory diseases. However, efficacy and safety remain the major challenges for turning gene therapy into a clinical reality. Several advances in vectorology have provided opportunities to address these issues including transductional and transcriptional targeting of viral vectors. The prior involves the modification of the virus tropism in order to increase the efficiency and specificity of target cell transduction. The latter comprises the use of cis-regulatory elements, such as promoters and enhancers (Fig. 1), to restrict transgene expression to specific tissues or patho-physiological conditions. Here we focus on recent developments and applications of endogenously-regulated promoter systems in gene therapy for autoimmune and inflammatory diseases, in particular rheumatoid arthritis (RA). © 2010 Springer Basel.