Gene Therapy Benefits and Potential Risks

Gene therapy
Gene therapy is an experimental method used to treat genetic illnesses by inserting healthy genes into cells, taking out the bad ones, or replacing a mutated gene. The idea of gene therapy rests on changing the internal structure of the DNA to prevent or cure illnesses such as Alzheimer’s and cancer instead of relying on drugs and outside treatment. Since its original conception in 1972, there were proponents and opponents of this method as both the benefits and potential risks  of this process were brought to the table.

There are two different kinds of gene therapy treatment: somatic cell gene therapy and germline therapy. The somatic gene therapy includes treating just the faulty genes by replacing them with the correct genes. By doing so, the body will then produce the right proteins needed to eliminate the gene causing the disease. This procedure merely corrects the recipient’s genes but cannot be passed down to his or her offspring.

With the germline therapy, however, it is possible to pass the corrected genes down to the next generation. This is done by having the gene inserted into the reproductive cells, so that the gene can be inherited by any future offspring.

As with any new procedure or issue, there are both benefits and potential risks that could arise with gene therapy. Gene therapy’s appeal comes when considering diseases such as Parkinson’s and cancer could potentially be fixed by inserting a healthy gene in place of the bad gene. It could also be a good option for diseases that have no established cure. The benefits that gene therapy could bring to the sick or dying are attractive, especially in the face of a life-threatening or incurable disease.

However, there are also many ethical concerns that come with gene therapy. As with any experiment that alters the genes, there are people who are concerned by scientists taking the role of God. Furthermore, since gene therapy is so expensive, it is only available to the elite or those who can afford it. If only the rich have access to being able to solve their genetic diseases, would this increase the gap of inequality?

The ability for this therapy to be properly regulated is also an issue.  Although the Recombinant Advisory Committee was established for the purpose of keeping gene therapy in check, there have been deaths and adverse side effects that went undocumented and unchecked by the government. The death of teenager Jesse Gelsinger by the gene therapy procedure caused the public to question the ethics behind the lack of transparency and accountability for his procedure.

Gelsinger, who had a rare liver disorder, decided to undergo the gene therapy with hopes of becoming better. However, the operation actually caused multiple organs to deteriorate and he died on Sept. 17, 1998. Before entering the experiment, Gelsinger was never adequately informed of previous patient side effects, as well as animals that have died during the experiment. In fact, the Food and Drug Administration suggests that this information might have been withheld from him; thus, he was not completely aware of what he agreed to.

The concern of the risks of the procedure itself remains another factor for skepticism. Operations require a huge amount of precision and accuracy, as well as complete research in order to administer the procedure correctly. The patient’s life could be forfeited if there is even one single mistake, as illustrated by Gelsinger’s death.

However, there has been a resurgence of hope in gene therapy due to a recent experiment done by the Cedars-Sinai Heart Institute in LA. They were able to temporarily regulate a pig’s heartbeat by injecting genes into a small portion of the pigs heart, thereby changing the ordinary cells in to cells that generated a regular rhythm. Further implications of this discovery is the development of a human pacemaker to regulate heartbeats, although Dr. Maraban the director of the Heart Institute states that it would take at least three more years to find a process that would be safe enough to administer to people.

In the recent years,  researchers have been able to help several patients who suffer from hemophilia by inserting a gene that helped them boost their body’s ability to clot blood. This experiment, reported in the New England Journal of Medicine in 2011, shows promising results to be able to stop excessive long-term bleeding in patients long-term through gene therapy.

Gene therapy is still incomplete, requiring a lot of trials and risks in order to find the correct solution. The benefits that gene therapy can bring to society is an ever growing prospect despite the potential risks that remain.

By Joyce Chu


NY Times
NY Times-Gelsinger
National Institute of Health

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