Gene therapy for rheumatoid arthritis: preclinical studies

Originally intended as a tool for the correction of inborn errors of metabolism, gene transfer technology has revolutionized the development of novel treatments to acquired diseases, such as cancer, autoimmune, and chronic inflammatory conditions. The concept of gene therapy for rheumatoid arthritis (RA) was proposed as an alternative to inefficient conventional drug delivery to the affected joints [1]. In this approach, introduction of exogeneous genes into the synovium should afford expression of therapeutic proteins directly at the site of the disease, thereby obviating the problems of inefficient systemic protein delivery and sparing the host from the side-effects associated with the systemic exposure to recombinant proteins [2]. However, despite its conceptual simplicity, many principal problems of gene therapy for RA remain to be solved before it becomes a routine clinical procedure. Here, I will summarize what we have learned from the preclinical studies using animal models, and discuss some unresolved issues of gene therapy for RA. These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.