Gene Therapy

GENE THERAPY 2

Overview on Gene Therapy

CHEM 120: Introduction to Chemistry

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Running head: GENE THERAPY 1

GENE THERAPY 3

Overview on Gene Therapy

Gene therapy is an experimental design that uses genes for the preventive or therapeutic measures purposes. In the nearest future, this approach/concept may allow health professionals treat various disorder by introducing a gene into a patient’s cells rather than using medications or surgical approach (Genetics Home Reference, 2020). Researchers are analyzing several concepts to gene therapy, comprising: Exchanging a mutated gene that causes disease with a healthier copy of the gene, deactivating a mutated gene that is malfunctioning and initiating a new gene into the body to help combat diseases. Although gene therapy is an interesting treatment proposal for a lot of inherited disorders, some form of cancer and certain viral diseases. This concept is currently undergoing experimental analysis only for deadly incurable diseases. However, the new gene can alter the way the cells behave by destroying the mutated version to modify its effects or by replacing a faulty gene. The main target of treatment in gene therapy are genetic disorders (Better Health, 2017).

Gene therapy is still in its experimental phase and health professionals are not yet sure of not only what all it can do, but what long-term effects treated patients may face. Some risks that have been discovered so far are cancer, inflammation and toxicity (NIH, 2018). One of the largest arguments and fears of gene therapy is the immune response towards the new gene. One particular patient named Jesse Geisinger became a victim in 1999 when he lost his life from complications due to an inflammatory response after his immune system reacted to the experimental procedure of gene therapy to treat a rare liver disorder (University of Utah). After Jesse’s death, a halt was put on gene therapy and the ethnical benefit to gene therapy was pondered. Though health professionals developed safer methods for injection such as giving the patient’s immune suppressants before the procedure or delivering viruses to cells outside the body, gene therapy was reconsidered and believed to be inhumane to experiment on humans. Some people wondered if it was right to alter an individual’s genes with such an unpredictable outcome while others considered the fact that due to the expensive nature of a gene therapy procedure, scientists may be developing a procedure that is only available to the wealthy (NIH, 2018). Members of the health team, while acknowledging Jesse’s death and the large expense of a gene therapy procedure, argue that gene therapy has many benefits and could be evolved in a way to benefit all people once more is known. A large benefit that consistently argued is if scientists could have the means to research and make gene therapy possible, the future may hold the ability to fix genetic diseases in developing embryos. If gene therapy were allowed to be practiced to success, medical practitioners could not only identify genetic diseases in babies but now take it a step further and inject a gene to cure the child so the baby would be cured and survive (Lewis, 2015).

However, curing unborn and newborn children is an exciting and groundbreaking advancement but some religious bodies worry of the power this advancement could bring. Besides their objection to the procedure on babies because they cannot give consent for such a risky procedure, religious bodies have concern this power could be abused to fix disorders which are not life threatening such as eyesight or even determine the physical traits of each child born , enhancing the population and mirroring the idea of “Hitler’s master race” (Rsrevision, 2015) . A summary from Moshier goes in detail of the possible misuse of gene therapy for human enhancement. “Major arguments against genetic enhancement are that it might reinforce irrational societal prejudices and it is morally wrong. If genetic enhancement is available, it can provoke “social prejudices against people who are obese, short, or mentally disabled” (Zallen A64). People who could not afford enhancement and those who do not wish to be genetically enhanced eventually might suffer discrimination (Moshier).

Health professionals acknowledge gene therapy could one day lead to the ability of parents to pick desirable genes for their unborn children however, they argue their interest in gene therapy is geared towards chronic and life-threatening genetic disorders. Furthermore, medical practitioners have placed their focus on ill patients whose quality of life has been completely overtaken by their illness. The first person to receive gene therapy was a 4-year old girl with severe immune -deficiency disease. Because her body failed to produce a vital enzyme, her doctor extracted some of her white blood cells, exposed them to a genetically engineered virus that invaded but did not destroy her cells and then transfused these cells back into the little girl. After the procedure, the little girl’s body began producing the proper enzyme and while she still needs follow-ups with her doctors, she is now living a relatively normal healthier life (Rocholl, 1996).

Medical scientist has had successes in gene therapy by curing disorders such as immune-deficiencies, hereditary blindness and blood diseases. They are optimistic that gene therapy could one day cure cancer, sickle cell disorder as well as other chronic conditions ranging from reproductive to organ disorder. Medical scientists also claim that the knowledge being gained regarding genes alone from their ongoing research is extremely valuable. They have advanced to an entire new age of medicine due to their new knowledge of genes. Medical scientists now better understand DNA, what causes hereditary disease and how to manipulate viruses so they will attack mutated gene or replace a defective gene to prevent or cure disease. If the science were allowed to advance, diseases that haunt many families would no longer have power over their loved ones lives and doctors may evolve into prevention of disease instead of treatment (University of Utah). While the benefits easily outweigh the risks for chronically ill patients, both critics and medical scientist admit the long-term effects of gene therapy on the patient’s life and the future line of these patients are unknown. While some patients may not be concerned with the long-time consequences due to an already poor quality of life, new findings are warning they need to worry of the effects it will have on their future children. Because gene therapy is designed to permanently replace a malfunctioning gene, there is looming risk the transferred gene may eventually rebel or pass on devasting mutations to the coming generations (Moshiers).

A further study in mitochondria implemented the use of stem cells where dysfunctional cells from women were infused with cells from healthy women. Known as the three-parent baby study, the cells were tweaked to live forever so scientists could continually mix defective genes with healthy ones from mothers to study the effects of cancelling out bad genes. During the study, the dysfunctional gene resurfaced 3 times. The study concluded that the original maternal mitochondria may eventually resurface meaning that any gene therapy experiment could go wrong and the problem can return (Neimark, 2017).

While gene therapy is still experimental, carries risks known and unknown, and may eventually fail in some patients, the benefits of curing some diseases permanently, learning about genetics in ways never possible before and easing the suffering of people with life-destroying genetic diseases is attractive and beneficial to medicine. The study of gene therapy could one day lead to a medical world where disease is prevented instead of treated. It is important to be ethical, human and sensitive towards all human beings during this research and the study of gene therapy but the research into the concept should not be completely shut down. We will never know that the therapy is capable of until scientists gain adequate knowledge into the pros and cons of gene therapy.

References

Genetics Home Reference. (2020, March 17). What is gene therapy? Genetics Home Reference. https://ghr.nlm.nih.gov/primer/therapy/genetherapy

Genetically Modified Organisms (GMOs) | Learn Science at Scitable. (2014). Nature.Com. https://www.nature.com/scitable/topicpage/genetically-modified-organisms-gmos-transgenic-crops-and-732/

ZAMEROWSKI, S. T. (2000, November). A Model for Integrating GENETICS into Nursing Education. Nursing and Health Care Perspectives, 21(6), 298.

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