Virus Vectors

Scientists are experimenting with viruses not only to distribute a needed vaccine but also to deliver healthy genes to cells with a genetic disorder, a procedure called gene therapy. Gene therapy could help patients who have a genetic disorder like cystic fibrosis or sickle-cell anemia, where only one or two missing or defective genes are involved.

In gene therapy RNA retroviruses are first rendered harmless. Then the healthy genes are inserted into the virus's genome. The altered virus is then injected into the tissue where it actively seeks out the appropriate cells to infect. As the RNA is copied by the cell's reproductive machinery, it is also incorporated into the cell's DNA.

This procedure is still in an experimental stage, but one of the first patients to benefit was an eighteen-month-old boy in England. He had a rare genetic disorder called severe combined immunodeficiency that prevented him from developing an immune system. He lived his first eighteen months in a plastic, sterile bubble room. Doctors removed bone marrow from the boy and used a virus to carry a new working version of the missing gene into immune cells in the marrow. The marrow was put back into the boy's leg, where it gradually started to produce healthy white cells that now protect the boy from infection.

One problem doctors must overcome is the unpredictability of where the new gene is inserted in the patient's DNA strand. If the placement is not exact, then the gene will not express itself. Scientists also have to battle with the patient's immune system, which seeks out and destroys these virus vectors before they get a chance to deliver their genetic package.

Even though gene therapy is still in the experimental stage, it has caused much controversy. Many people are concerned about the safety of the procedure as well as the long-term effects of inserting a virus, even a disabled one, into a person's body.

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