How Gene Therapy Works

Gene therapy is a scientific process where abnormal genes are replaced by either normal genes or altered genes to try to correct some kind of hereditary problem that a person was born with. In some cases gene therapy has proven to be a wonderful thing, while in others, gene therapy has proven to be more dangerous than anyone could imagine.

As there are more studies done on the body, scientists have found ways to determine which genes in the body cause certain types of diseases. Things such as cystic fibrosis, multiple sclerosis, blindness and cancer are considered as things that are possible to correct with proper gene modification.

In a couple of recent reports, gene therapy is credited with helping one little girl overcome what’s known as “bubble boy” disease (ADA-SCID), which meant that she had the antibodies to protect her from disease initially, but after the reintroduction of stem cells from her bone marrow, which were exposed to an engineered retrovirus, had been injected back into her body, her immune system became populated with functioning T cells, which restored protection to her body and allowed her to be able to go outside and play like any other child.

For another patient who was suffering a progressive loss of vision from a disease known as Leber congenital amaurosis, he had a gene known as RPE65 injected into his retina, after being exposed to a virus known as adeno-associated virus. In less than a week his eyesight started to improve, and he was able to see more light which made it easy for him to be able to walk around in the evenings. He also was suddenly able to see stripes on his clothes, which he hadn’t been able to see for years.

Although these kinds of results, which were dramatic, were expected, even though not as fast, there have been some failures which have resulted in death. Sometimes putting a gene back into someone’s body activates the white blood cells to such a degree that they overwhelm the system and kill the host. It’s for this reason that gene therapy still is considered on the fringe of normal medicine, and is still considered to be in experimental stages. Still, the possibilities are endless for treating all sorts of diseases in the future.

See more:
Gene Therapy Pros & Cons
Gene Therapy Journal
The Ethics of Gene Therapy