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Collectively, autoimmune diseases constitute a major, unmet, clinical challenge. Although no single autoimmune disorder is highly prevalent, there are over 80 of them, and 20% of the population is affected; approximately 75% of patients are women. Because these diseases are generally incurable and difficult to manage, there is a pressing need for novel approaches to their treatment. For reasons detailed below, we have proposed that gene therapy merits investigation in this regard.1
Traditional pharmacological approaches to treatment entail the synthesis of small, diffusable compounds given orally or by injection. These approaches have yet to provide ideal agents for use in autoimmune diseases. Recent research, however, has identified a number of proteins with the potential to improve treatment, but these are difficult to administer long term. Gene transfer provides the opportunity to deliver protein products, as well as therapeutic species of nucleic acids, such as antisense RNA, much more efficiently than traditional methods of drug therapy. Furthermore sustained, in situ production of the gene products would eliminate the need for frequent re-administration. In addition, gene delivery and subsequent expression has the potential to be highly localised, if needed. Indeed, gene therapy’s greatest strength may be its ability to produce high, sustained concentrations of therapeutic macromolecules within a defined anatomical location. In the case of RA, for example, anti-arthritic proteins are already being delivered systemically to patients by subcutaneous or intravenous injection. There is, however, no practical method for transferring these molecules selectively to joints in a sustained fashion.
In its broadest sense, gene therapy is the transfer to patients of a gene, or genes, for therapeutic purposes. With some exceptions, such as skeletal muscle and skin, naked DNA is not efficiently taken up and expressed by mammalian cells. For this reason it is necessary to use vectors as agents of …