The Potential of Gene Therapy

Photo Dredit : Svilen Milev

Gene therapy is a revolutionary treatment that has the potential to become the medicine of the future. Gene therapy is the process by which scientists can replace faulty genes and replace them with more predictable and preferred genes. For example scientists have developed a way to mutate genes in order to make them immune to Malaria (“Splice of Life”). Scientists have also advanced this technology as far as editing complete embryos. From this they are given a preferred germ line. Germ line is another way to say the cells responsible for reproduction, or sperm and eggs. When this embryo develops, and then later reproduces, it also will pass on the preferred germ lines that have been altered. This is significant because babies will not have to deal with the devastating effects of genetic diseases passed on to them from their parents. Doctors can improve the overall quality of life for the human race. Essentially, doctors are improving the overall quality of life for the human race. However, Nature has an article mentioning that the recent success in the evolution of gene therapy could take a turn for the worse, specifically- “gene therapy might creep beyond eliminating deadly or debilitating heritable disorders to include disabilities, less serious conditions, and cosmetic and other supposed enhancements — leading to ‘designer babies’ and raising the spectre of eugenics.” (A Splice of Life). This is a stretch considering the author failed to cite any research of this being feasible to solidify his theory. The bottom line is that with a technology as advanced as gene therapy there will always be questions as to where the limits should be. Gene therapy is a growing medical field that still needs support from big health organizations such as the World Health Organization. There are also ethical and legal concerns surrounding gene therapy such as how far is too far when editing a genome. Medical researchers need continued funding because the potential upside outweighs the negatives.

Nature also mentions just how much power gene therapy has. Not only can it fix genetic diseases that a patient is given from its parents, it can also alter the patient’s gene line so that it is completely immune to things such as malaria or HIV (Cell Stem Cell). This type of research is the future and I completely agree with the author in this case. The author even goes as far as to say “A total ban on research would therefore seem counterproductive”.

Nature seems to then track back in its steps when it claims that there are currently better alternatives to gene therapy. “Preimplantation genetic diagnosis and selection of healthy embryos during in vitro fertilization already provides a safer alternative for avoiding genetic disease in newborns — as can prenatal screening and abortion” (A splice of life). The claim about abortion is completely false. Aborting a baby that has a genetic disease is not as effective as treating it because abortion is preventing it from having a disease free life. Also the previously listed treatments, in my opinion, are most likely as developed as they’re ever going to be. Gene therapy still hasn’t hit its ceiling, meaning there is a slew of untapped potential in this research. “While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients with oral diseases” (Baum 115-118).

In regards to regulation of gene therapy Nature claims that “national governments need to step up on this issue” (A Splice of Life). Regulation is important to the progression of gene therapy. Not only does it provide safe and ethical guidelines, but it also makes it more acceptable in the public's eye. The “WHO” or “World Health Organization” is composed of scientists, regulators, and ethicists. They meet to discuss topics such as whether or not gene therapy is ethical. This regulation is important because it can set guidelines for the entire planet. It can also set the general guidelines for each country to base their research off of. The article lists other things the WHO has met to discuss, such as Ebola. WHO met rapidly last year to discuss the ethical concerns of the Ebola vaccine and just how safe it was for general use around the world. Overall Nature does a good job describing the WHO because it provides specific details that were left out of previous claims made by the author. For example when the author made the claim that the world will eventually see “designer babies” they provided no facts or sources to validate their claim.

The ethical problems surrounding gene therapy can be avoided through monitoring. In theory nothing can get out of hand when the proper measures are being taken and those responsible are held accountable. (Baum 115-18).As said by Nature, “Each new application of a gene therapy to humans undergoes extensive and multi step approvals that are required to demonstrate ample proof of both efficacy and safety in animal models.” The right steps are already in place for animal testing.The hope is that they can be easily rolled over into human use. Also no animals are hurt when developing, or being treated with gene therapy. This should silence any critics that say this treatment could be unethical. Science Direct also makes the same stance. They believe that gene therapy is ethical because it has been tested thoroughly on animals and yielded no negative results. If a patient is immune to the therapy, they will simply provide a transplant and that will solve the issue. They make the transplants safe by covering them in specialized stem cells that the recipient’s body won’t reject. They also believe the same exact procedures will roll over into use with human patients (Cell Stem Cell). This will make the transition to human treatment quick and painless. They also don’t harm other living things to acquire these transplants. The Nature article claims that, as of yet, gene therapy isn’t very safe or predictable, but the other two sources I viewed stated differently. The Nature article failed to mention the success researchers have had in applying gene therapy to animals. Science Direct and The Online Library prove that Gene therapy is ethical because of the success it has had in animals when curing diseases while simultaneously causing them no pain. They also describe how detailed the process of approving gene therapy for use is. In Science Direct alone the process took more than a year to get approved. This shows just how much administrators look into the process of gene therapy. If anything was unethical, it wouldn’t have passed such a rigorous examination.

Gene therapy is something that should be legal to practice. As previously stated, the ethical concerns about gene therapy are non existent. The benefits of gene therapy heavily outweigh even the most ridiculous of claims. Nature states that “an entire ban on gene therapy would be counterproductive”. The author doesn’t have the realization that gene therapy is an important asset to the advancement of the medical field. They also claim it isn’t very reliable. This may be true for the specific study that they looked into, but other studies with different focuses have found gene therapy to be very efficient. Researchers have had very admirable success rates for a treatment that is not quite fully developed. When applying gene therapy for oral diseases to humans “the AdhAQP improved salivary flow rates in the targeted parotid glands of six subjects and led to a reduction in subjective complaints in five of those individuals.” AdhAQP is a vector used to replace DNA in a sequence (Baum 115). Success in five out of six patients is an impressive feat in any trial. They also tested their vector on rats. More than 200 rats were given a vector to test. What a vector primarily does is find the place of the DNA it is suppose to target, and then remove it. Once it has removed the target sequence of DNA it replaces the gap with DNA that is was carrying. None of them showed noticeable side effects. This proves that the way in which the treatment is delivered is also safe. If this treatment were made illegal, then researchers wouldn’t have advanced it as far as it is today. This is a prime example of why gene therapy should be legal. If the government were to cut gene therapy right now, then they would be hurting the future of medical development. The government needs to at least let gene therapy progress and let it fully develop to its full potential before imposing a complete ban. Nature is right to say that that gene therapy shouldn’t be banned, but they’re undervaluing its potential upside because stopping the development of futuristic medicine would be a very lousy decision.

Gene therapy is worthy of development because it can eventually become the ultimate medicine. In some fields, specifically oral diseases, it has become the preferred treatment because of its effectiveness and efficiency (Baum 115). The Online Library describes just how productive it has been. If they continue to develop then the sky becomes the limit. The eventual goal is to be able to translate all of the successes they have had in treating animals into treating humans. Given the success in oral treatments alone, gene therapy should be refined, a statement in which Science Direct also agrees. Imagine living in a world where HIV is no longer a problem. Gene therapy is worthy of development just because of its potential (Cell Stem Cell). Using the research done in “Cell Stem Cell” by Science Direct on non human primates, scientists feel they are comfortable now to begin testing on humans. Cost is also something to consider when talking about investing in gene therapy. The price of lifelong treatment for those who currently live with aids can be very expensive. If the patient was cured using gene therapy, they wouldn’t have to have to pay bills every month for their treatment considering It would just be a one-time procedure. As of right now, gene therapy is very expensive considering materials have to be purchased and researchers must be compensated. . None of the companies turn a profit to support themselves and many research groups have to look for outside donations.

In closing, gene therapy is a revolutionary treatment that needs continued support. It has the potential to completely change the way we treat diseases. The potential ethical concerns from the public aren’t anything that should be taken seriously. With the right precautions and guidelines in place, gene therapy won’t go as far as creating “designer babies.” Gene therapy is the future of medicine and needs to be treated as such.











Works Cited:




"Splice of Life." Nature 521.7550 (2015): 5. Web. 1 Oct. 2015.


Kiem, Hans-Peter, Keith R. Jerome, Steven G. Deeks, and Joseph M. McCune. "Cell Stem Cell." Science Direct 10.2 (2012): n. pag. Web. 1 Oct. 2015.


Baum, Bj. "Gene Therapy." Oral Dis Oral Diseases 20.2 (2013): 115-18. Web. 1 Oct. 2015.