GENE THEIFAPY IN TREATMENT OF RHEUMATOID ARTHRIT AND OSTEOARTHRITIS

Abstract

Over the past decade, dramatic advances in molecular genetics science have provided new insights into fundamental mechanisms of health, and disease. Not only have genes been discovered that underlie inherited rheumatoid arthritis. There is also increasing appreciation of how these genes maintain physiologic homeostasis and how they contribute to pathologic conditions such as rheumatoid arthritis.

A minimal definition of gene therapy is the transfer of genes to patients for therapeutic purposes. Its most obvious value lies in treating genetic diseases, such as Gaucher disease, where the aim is to compensate for a single heritable genetic defect. However, genes can also be used to treat acquired diseases. In the latter context, the gene is utilized as a biologic system for the delivery of gene products, both RNA and protein. In most rheumatic diseases, genetic influences are polygenic, complex, and quantitatively less important than environmental influences. For these reasons, the main application of gene therapy to these disorders will be initially ass a means of delivering therapeutic gene products.

The concept of gene therapy for arthritis is that genes may be administered to patients that enable cells within the body to express therapeutic gene products. Gene therapies aimed at introducing genes into the body to reduce local inflammatory processes, control cellular proliferation, promote regeneration or repair of damaged tissue, or even re-engineer the function of the immune system.

Delivery of medication to synovial joints by conventional means is inefficient. Direct intraarticular injections have the disadvantage of rapid clearance by the lymphatics of materials reaching the joint space. Systemic injection requires high doses to deliver effective concentration of the medication in the joints, exposing other tissues to the high