Gene Therapy

Gene therapy is a form of treatment that doctors and scientists hope to use against genetic diseases. Instead of battling a foreign body, which is not the source of problems in genetic diseases, the goal in gene therapy is to replace faulty genes causing illness in patients with correct ones. What one uses to replace the old gene with the new one is called a vector. There are many different vectors for placement of new genes, such as using viruses, naked-DNA injection, dendrimers, and others.

The first approved gene therapy procedure was performed in 1990. It was meant to treat severe combined immunodeficiency, or SCID, a genetic disease in which certain receptors on white blood cells are not correctly coded for in the corresponding gene, rendering white blood cells useless. Doctors removed white blood cells from the patient, a four year old girl, let the cells develop in cultures, then inserted the correct gene into the cells and put the cells back into the patient. Even though the procedure only lasted a couple months, it boosted the patient's immune system so much that she could now attend school, which she previously couldn't do due to her extremely high risk of fatal infections from what are considered mundane conditions, such as colds. This case shows promise for gene therapy, even if results aren't permanent.

http://en.wikipedia.org/wiki/Gene_therapy
http://en.wikipedia.org/wiki/Severe_combined_immunodeficiency