Gene Therapy

Drugs in development for Rheumatoid Arthritis
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Used here with permission

Gene Therapy

Genes are sections of DNA that express traits by coding for specific proteins. Research has traced the underlying causes of many diseases to defective genes and problems in gene regulation. Technical advances allow for new genes to be introduced into and accepted by living organisms. This has led to gene therapy: the insertion of healthy genes into the body in order to compensate for genetic defects. Gene therapy is being tried as a method of treatment for a range of diseases including cystic fibrosis and a number of diseases caused by enzyme deficiencies. Initial results from these attempts have been encouraging.

There is no clear, single genetic defect that leads to rheumatoid arthritis. It is thought that RA is a result of both genetic and environmental causes. There is increasing evidence that a number of inflammatory proteins produced by the genes of immune cells have an important role in the origin and development of RA. Interfering with these cells may disrupt the process of RA itself. This could take place by decreasing the molecules that cause inflammation and increasing the molecules that reduce it. A great deal of research is ongoing to design safe and efficient genes to interfere in the inflammation process and to develop methods of introducing them into the body. 

Previous research has been successful in introducing inflammation-modifying genes into the lining of joints in mice and rabbits. These advances give hope for intervention into the genetic causes of inflammatory diseases. There are now many gene therapies in clinical development.