Gene therapy is a form of treatment that mutates the patient’s DNA to produce certain desired effects. This is done by extracting his or her DNA from the nucleus of the cell, manually changing the genetic information, and inserting this new DNA back into the patient. The benefits of using this treatment is that it can completely reverse the genetic disorder, which improves the overall health and wellbeing of the patient; however, Gene therapy in zygotes, or a fertilized ovum, is unnatural and causes negative impacts not only on the individual, but society as a whole. This collective issue raises scientific, ethical, and political concerns. Gene therapy should be available to adults and children, but should not be available to prospective parents. 

Gene therapy first started to spark interest when genetic research was primarily focused on prokaryotic, or single-celled, organisms; molecular biologists in the 1960’s built the foundation for modern medicine by exploring the mechanics of genes, such as its contents, its formation, and its expression. In the last 50 years, both science and technology have greatly advanced. Today, there are not only studies on the genetics of eukaryotic, or multi-celled, organisms, but society uses this research for clinical application to correct genetic deficiencies (Judson).

Gene therapy is achieved through the use of genome editing, or changing the sequence of DNA. The development of CRISPR-Cas9 technology, which stands for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, is what enables genome editing to take place. This works by targeting a specific DNA sequence in the genome and then creating a complimentary RNA strand in the lab. This new, man-made RNA then binds to the Cas9 enzyme and is then inserted into the organism. Once in the organism, this RNA/Cas9 complex enters the nucleus of the cell and searches for the programmed DNA. Cas9 then unzips the DNA at a specific part and matches it to the RNA. When complete, Cas9 then cuts the DNA and the cell begins to repair itself to put the new DNA back together. With the new

DNA sequence, the function will then continue to function using this new genome (Zhang).

Ideally, gene therapy can improve the health and overall quality of life of an individual. For instance, someone with Alzheimer’s, a disease that decreases cognitive functions and causes memory loss, can manage their symptoms more effectively by using this treatment. Comparatively, leukemia, cancer of the blood cells, can be combatted by extracting specific cells from the patient, altering its DNA, and placing it back in the patient to target and kill off the remaining cancer. Gene therapy, however, is a last resort for them and is only considered when all alternative treatments have already proved ineffective. Gene therapy should still be used, but with extreme caution. If successful, a patient’s life could be changed for the best and, in some cases, the condition could be reversed, potentially terminating genetic diseases entirely. When completely removing the possibility of someone developing a genetic disorder, one also eliminates the chance of that trait being passed down to the offspring. 

Alzheimer’s disease (AD) is a form of dementia that causes memory loss, mood swings, and confusion in those affected. Amyloid-beta peptide, a protein found in patients with AD, causes brain cells to degenerate. The formation of this protein is caused by a specific gene that triggers the production of plaque to build up in the brain. When these brain cells die, they cannot regenerate but symptoms can be combatted with a combination of pharmaceutical and alternative therapies. However, when these treatments are inadequate, as a last resort, gene therapy can be utilized by using CRISPR-Cas9 to target the gene that causes the formation of amyloid-beta peptide proteins. This will stop the cells from being terminated, therefore ceasing the symptoms. (“Could Gene Therapy Work for Alzheimer's Disease?”). The patient would not be able to regain lost memories, but it could stop more damage from being done.

Cystic Fibrosis (CF) is a genetic disorder that causes mucus to develop in the lungs, which induces infections in the lungs and pancreas (Lindee and Mueller 318). According to the Cystic Fibrosis Foundation, symptoms include salty-tasting skin, coughing with phlegm, frequent lung infections, shortness of breath, wheezing, and increased bowel movements. In 1989, researchers determined that the gene initiating this build-up of mucus was the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The normal function of CFTR is to present the blueprint of a protein that serves as a medium for chloride ions to enter and exit epithelial cells. These cells produce bodily fluids such as sweat, tears, saliva, and mucus. This is important because mucus provides lubrication and protection for internal organs. However, in CF, a mutation in the CFTR gene causes the instructions for the protein channel to be inadequate. Without a properly functioning pathway for chloride ions to move in to and out of the cell, the cell begins to make abnormal mucus that restricts the movement of air within the lungs, which causes mentioned symptoms. Meaning, the correction of the mutated CFTR gene will reverse these effects. (“CFTR Gene - Genetics Home Reference.”) Ever since the early 1990’s gene therapy has been the most promising treatment for CF since it does not merely cure the symptoms, but it repairs the origin of the problem. However, the research of gene therapy to treat CF has staggered in recent years; initially the first clinical trials resulted in adverse and fatal effects, causing unpopularity in political and public opinions.

Although there is controversy of gene therapy to treat diseases in adults and children, there is far too much potential for it to be left ignored; research must restart and continue in order to provide patients with safe, effective treatments. Not only will corrections of genetic disorders benefit the patient though; it will impact future generations to obliterate these diseases as a whole.

Due to scientific advances in the medical community, there are now more options than ever for treatment of fetuses developing in the womb. Such options are stem cell transplantation, invasive fetal intervention, open fetal surgery, and gene therapy. These therapies can correct a wide variety of conditions, such as anemia and immunodeficiencies (Phithakwatchara). However, it is the responsibility of medical professionals to fully inform and properly advise the parents involved of the risks and benefits of these treatments; effective strategizing of doctor-to-patient interactions is critical for all parties involved. Since this technology is new and still improving, some effects are unclear and irreversible. More research is necessary before any clinical applications can be performed. Fetuses with genetic disorders used to have limited options for therapies, but stem cell research has shown progress toward the possibility of using genetic transplantation as a form of treatment; however, there is seldom clinical application since there is insufficient evidence of success in these trials and unsuccessful results go unpublished (Phithakwatchara). 

Familial counseling for fetal gene therapy is a critical step in the treatment process. Medical professionals must thoroughly explain the science behind the therapy as well as the possible outcomes to all parties involved. However, since most are unfamiliar with this jargon, it is the professional’s responsibility to effectively communicate to the patients with language they can understand. For instance, prenatal therapy expert Phithakwatchara urges doctors to recognize all aspects of the patients’ lifestyles, rather than just focusing on one, stating that “familial, societal, cultural, and economical factors must be considered for the family to reach an informed choice”. The best way to prepare doctors for these various interactions is to provide them with training that is specific to each person.

The main approach for prenatal medicine is to treat the fetus as a patient, rather than a person. This allows for the team involved to treat as standardly as possible and not become so caught up with the possible outcomes. The team is expected to uphold the standard of care, educate the parents involved, respect the decisions of the parents, and take into consideration the health of both the fetus as well as the mother. Doctors also know when to offer termination of the pregnancy, which could be a possible option if the treatment would result in chronic disability of the fetus; however, it must be at a certain term in the pregnancy before the woman’s options become restricted due to legal dispositions.

In germline gene therapy, sex cells can be modified to heighten natural features like height, athletic ability, and physical appearance. These alterations of egg and sperm cell’s DNA impact the offspring (Hanna). While it may avoid typical genetic disorders, it may result in these individuals encountering new man-made genetic diseases. Not only does this involve this individual’s health, it interferes with their right to choose; it strips them of their right to decide which treatment option is personally best. Since the rights pertaining to a potential citizen of America are not explicitly described in the Constitution, Congress must now develop laws and regulations of gene therapy to protect those rights. Even though the Food and Drug Administration (FDA) has standards set in place for the treatment to be administered, there is no definite limit of when this treatment becomes too extreme. The government would have to determine what is considered ethically acceptable or not. Because of these potential problems, the government should not allow parents to take part in this therapy.

Researchers argue that gene therapy can become ineffective; the body can build a resistance to the new genetic information, which causes for additional treatments to become necessary. Consequently, if too many treatments are provided, the body can trigger a mechanism to defend against this new foreign antibody, which in turn causes the body to attack itself (Mandal). The possibility of adverse effects is the motivating reason behind the termination of gene therapy research. For instance, in the early 2000’s two children developed leukemia in a clinical trial, causing a stirring in the medical community. Shortly after, in 2009, a teenage boy died due to organ failure as a result of another clinical trial. After these events, the government terminated funding to cease it from reoccurring.  Because of overselling to investors involved in the gene therapy industry, the public perception of its progress was skewed, leading most to believe that research was improving more than it actually was (Lindee and Mueller 325-326). Because of this inconsistency in the communication of results, there was a cycle of clinical trials being halted and then reinstated again. 

Agreeably, every treatment has its risks and they should be considered before starting the treatment round. However, if the appropriate precautions are set into place, the chances of the patient experiencing extremities is rare and if they do, it is minimal.  Immune system deficiencies can even be combatted with other medications. Most diseases only require minimum rounds and if the patient is financially able to do so, they should have the option to complete as many rounds of treatment that is safe. Also, an abundance of progress has been made in the technology used to administer this treatment. With all of the scientific breakthroughs including discoveries of new genes and the development of CRISPR-Cas technology in the past decade, scientists are more equipped and knowledgeable than ever before.

There is reason to believe that gene therapy in all of its forms should be avoided. One argument is that changing the human germline, or DNA blueprint, can cause catastrophic effects of future generations; the slightest genetic defect will be passed on from parent to offspring, causing a whole new breed of human. With the introduction of man-made DNA, new proteins can arise as well.  With these proteins, however, scientists can create new functions for not only humans, but in animals as well. The formation of these new proteins can especially be beneficial in the field of synthetic biology (Wang).

 Foht, an associate editor of The New Atlantis: A Journal of Technology and Society, speaks on the behalf of conservatives, making claims that encompass the group as a whole. He states that as a Conservative, it is imperative to understand that the family is the structural foundation of society; by removing these genetic defects from the human genome, scientists in turn “put an abstraction, the human gene pool, above that fundamental unit of society, the family” because they prioritize “genetic perfectionism” (Foht). Meaning, to tinker with genetic engineering is to disregard the value of the family unit. 

However, most parents who decide to perform gene therapy on their child do it out of the concern for the child’s wellbeing. The parents who make great financial sacrifices for these medical procedures, have the interest of their child at heart; they aim to provide a better quality of life. Most parents take advantage of clinical trials because they are desperately searching for any remedies. If anything, this attitude shows a prioritization of the family as well as the child.

Gene therapy should be available to adults and children, but should not be available to prospective parents. It has the potential to benefit adults with AD and CF as well as children with Leukemia, but should only be reserved as the final remedy. Fetal gene therapy should be avoided until there is more evidence of success and the ethical and societal dilemmas are resolved; there is too many complications involved for this treatment to begin. Regardless, research needs to be prioritized and continued in order for clinical trials to take place. When approached with extreme caution, gene therapy is a useful treatment with a promising future. 
