Death is a sad, yet unavoidable part of life. Everyone has experienced, or will experience, the death of a loved one. It is a rough thing to go through. Often times it is felt that the deceased was whisked away too soon, or that they had more to live for. These thoughts could someday become preventable, as the diseases or conditions that affected the deceased become preventable. This may be achieved through gene therapy. For people with genetic disorders or acquired conditions such as cancer, the chance to live a full, complete lifetime is closer than most think. Gene therapy is in its early stages, but has already helped dozens of people with illnesses that were previously thought incurable. Gene therapy is a good medical treatment technique because it could prevent hereditary illness and disease, eventually eradicate such diseases, and allow individuals that would be born with hereditary diseases lead better, more normal lives.

     There are two different types of gene therapy, somatic and germline. Somatic gene therapy is quite ingenious, and has two different forms. During in-the-body gene therapy, bioengineers modify an existing virus, usually an adeno-associated virus. This is a type of virus that is not known to cause any disease in humans. The engineers then add in synthesized DNA designed to produce a specific reaction within cells. The virus is injected into the patient, and it attacks the patient’s somatic cells (non-sex cells). These cells have specific genes removed by the virus, and the virus then implants the synthetic genes into the host cell’s DNA. The faulty genes have now been removed, and the proper genes in place.  This cell will start acting as it should, then multiply and replace the dysfunctional cells. In addition, the virus will reproduce, causing faster spreading of the proper genetic code. In-the-body gene therapy is the choice for patients with hemophilia, as adeno-associated viruses attack the liver and blood first. This can effectively spread non-hemophilia genes quickly to areas associated with it. (Arthur Neinhuis)

     Out-of-the-body somatic therapy is different, but effective in its own way. Doctors take either blood or bone marrow from the patient, and then isolate immature cells. In these cells it is easier to modify their own DNA, as opposed to adding new DNA, due to their early developmental stage. The cells are placed back into the patient’s bloodstream, and make their way back to the bone marrow (where blood cells are manufactured). These fixed cells replicate and edge-out damaged cells, and correct the deficiency or disorder of the patient. Out-of-the-body therapy can also be achieved through the use of retroviruses; HIV is an example of a retrovirus. Retroviruses are highly specialized in replacing host DNA, so genes are easily implanted into the patient. However, retroviruses are also able to place their genes anywhere in the host DNA, which could possibly damage the patient even further. This treatment has been very successful in treating Severe Combined Immunodeficiency. Children with SCID have no immune system, and can die from the most insignificant virus or bacteria. Among those treated for SCID, gene therapy has been successful in over 90% of cases; however, five children developed leukemia, but four have been cleared of it. Doctors are now in the process of refining delivery systems in order to place genes in the correct places, and somatic gene therapy looks very promising. (Arthur Neinhuis)

     Human germline genetic modification (or HGGM) is a highly controversial treatment that has not yet been attempted. In HGGM, a patient introduces specific genes or a specific gene into either their eggs or sperm. This type of gene therapy is meant to permanently alter future generations’ DNA. In most cases, this would be used to prevent the presence of hereditary disease, like cystic fibrosis, in offspring.

     In the case of HGGM, it would be possible to prevent and even completely eliminate hereditary conditions like Tay-Sachs disease or hemophilia. Genetically-passed diseases such as these are contained within the genes of the parents. The parents may not be affected, but the genes could be passed on and activated in the offspring. Future parents may be able to undergo HGGM and erase the disease-causing genes from their gametes, or eggs and sperm. This would allow the offspring to be born free of the possibility of having an inborn condition. Not only would this technique be able to prevent the disease, it could eliminate it all together if the DNA specific to certain conditions disappeared over time. Tens of thousands more children would be born healthy and normal each year.

     Patients that undergo somatic gene therapy can also lead much happier and more normal lives. In 2007, doctors led by Philippe Leboulch of Harvard Medical School used an engineered virus to insert a section of DNA into an unnamed patient with Beta-thalassaemia. This disease is a type of anemia that affects the blood’s ability to carry oxygen, leading to possible organ failure and death without regular blood transfusions. As of June 2008, the man received his first transfusion in over a year, and was reported to be in good health. (Physorg.com) Success like this is what drives gene therapy science forward.

     Of course, this experiment is just one of many attempts at gene therapy. These trials come with limited and widely varied amounts of success. Many of the arguments against gene therapy would ask if the risk is worth the reward. The most famous failure with gene therapy is the story of Jesse Gelsinger. Gelsinger was born with OTC deficiency. This results in the inability to break down ammonia, a natural byproduct of metabolism, and leads to death in about fifty percent of cases. In 1999, Gelsinger, only 18, underwent a gene therapy trial conducted at the University of Pennsylvania. Within only hours of receiving his dose, he became ill. Four days later, he died of massive organ failure due to an extreme overreaction of his immune system. (UVM.edu)  In this instance, the researchers should have been more cautious about administering safe dosage; but this doesn’t mean that gene therapy as a whole is a failure. It is done by trial-and-error, like any science or technology.

     There are other issues that are raised by anti-gene therapy groups. According to Chris Wharam, the two main concerns are “playing God” and “the slippery slope.” Of course, with any new scientific advancement comes the concern that we’re stepping outside our natural abilities as humans. Gene therapy has received specifically critical reactions like this because treatments are designed to actually change our genetic code. These concerns are focused mainly on HGGM, declaring that having a hand in shaping our children before they are even born is unnatural, and should not be allowed. One easy counterargument though is that God made the technology available, so this is not going against His will or acting as Him. Another, more viable aspect of the “playing God” argument against gene therapy is that eventually we will be able to change things about our children such as physical appearance, intelligence, or behavior. This is a cause for concern, as the thought of “superhumans” with intelligence like that of supercomputers and the physical qualities of Olympians traipsing around being better than most is unnerving. I’m sure, however, that as gene therapy becomes increasingly advanced, it will also become increasingly regulated by the NIH and FDA, preventing such occurrences.

     The “slippery slope” argument pertains to the misuse of gene therapy, or using it for things other than its intended purposes. Wharam cites an experiment in which researchers are attempting to genetically alter a chemotherapy patient’s hair follicles to reproduce, so cancer patients won’t have to go bald. He then goes on to say that this therapy could eventually prevent or reverse baldness in non-cancer patients. Wharam then says that, “This example is quite petty, but those against gene therapy cite it as just the beginning in a "slippery slope" towards the development of a superior human.” (Wharam) People could eventually use gene therapy for a wide range of things that have nothing to do with the reasons they were created. Just like the “superhuman” argument, however, I am sure gene therapies will be heavily regulated as their use becomes more commonplace.

     When done carefully and correctly, gene therapy can cure disease, prevent disease, and, since there will no longer be disease, make lives easier and more enjoyable. Clearly there are risks, but there are risks to any science. In my opinion, scientific advancement should be a welcome and useful thing in any form, because this can lead to more advancement. In order for gene therapy to be a successful medical tool, people need to cast off the shackles of fear, and embrace science. It can be a much better world this way.