Gene Therapy

Drugs in
development for Rheumatoid Arthritis


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

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.