Gene Therapy

This means introducing an active gene into the body's cells to compensate for the inactivity, or inadequate activity, of the person's own gene. Preparing replacement genes has been successfully managed, but devising an appropriate method of delivery into the appropriate cells of the body is a difficult technological task that has limited the application of gene therapy. As with all transplant procedures, immune reactions by the body are a significant issue to be managed for a successful outcome.

Trials of gene therapy have demonstrated the success of these techniques in a number of animal models of various disorders, and experimental trials have demonstrated success in human patients with Haemophilia A and Severe Combined Immuno-Deficiency.

Gene therapy has the potential to correct conditions that affect the brain and the central nervous system, and this has implications for a number of diseases such a Parkinson's disease and Alzheimer's, as well as for a number of conditions where the incorrect gene expression leads to intellectual disability.

Alternative methods of gene therapy are also under investigation. This includes trying to develop methods to activate inactive genes, or to correct overactive genes, without introducing foreign genes. This concept is known as gene repair, but is at experimental stages only.

A considerable amount of research is going on into ways of perfecting the technical issues so that gene therapy may be applied to a wider range of diseases and disabilities.