Gene Therapy

Gene therapy is an novel technique that uses genes to treat or prevent disease, including inherited disorders, some types of cancer, and certain viral infections.

Currently, the therapy is considered "experimental", and it is currently only being tested for the treatment of diseases that have no other cures.

However, in the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

The techniques involved in the therapy include:

• Replacing a mutated gene that causes disease with a healthy copy of the gene
• Inactivating a mutated gene that is functioning improperly
• Introducing a new gene into the body to help fight a disease

The first successful gene therapy trial was conducted in 1997, when scientists successfully used gene therapy in humans for growing new blood vessels to bypass plaque-clogged arteries in the legs of 16 people (out of the 20 experimented on).

In the therapy, called therapeutic angiogenesis, the gene was grown in the lab, then injected into a patient's leg muscles, making existing blood vessels to generate new blood vessels.

Also, in related field of research, following the discovery of longevity-related genes, researchers have been trying to find drugs to activate them.

Because this is an experimental field of anti aging therapy, many safety issues are still under investigation, including the fact that some studies have suggested that the current gene therapy methods may pose an increased chance of causing cancer.

Gene Therapy - Studies

Ginn SL, Curtin JA, Kramer B, et al. Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia. Med J Aust 2005; 182: 458-463.

Balicki D, Beutler E. Gene therapy of human disease. Medicine 2002; 81: 69-86.

Thrasher AJ. Gene therapy: great expectations? [editorial] Med J Aust 2005; 182: 440-441.


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