Gene Therapy Analysis

Junior (College 3rd year) ・Biology ・APA ・4 Sources

Gene therapy can be defined as a procedure for enhancing or treating medical condition thru genetic modification of the cells of the patients. The technique lets in treatment of acquired and inherited sickness through deliverance of a therapeutic gene material collectively with related regulatory substances into the nucleus to right function loss as a result of mutation or to assist gene product with deficiency at physiologic levels. Gene therapy comes with crucial troubles for social policy, public health, and medicine concerning the occasions that would need its application, the way of implementing it and its impacts. Questions are raised regarding whether people need to be allowed to refute or accept its implementation or if it needs to be a need to just to be a must just as the screening of new babies is in some regions. Another issue raised is whether individuals should control access to the consequences of gene therapy and whether the results can be revealed to third parties like insurers or employees. This article will be discussing among these issues and provide the opinion of the author.

Keywords: gene therapy, disclosure, new born screening, gene transfer.

Gene Therapy

Gene therapy can be defined as experimental techniques which utilize genes in treatment or prevention of illness (Maeder & Gersbach, 2016). Among the approaches employed in gene therapy is the replacement of a gene that is mutated and causing illness with a stable and healthy gene, inactivating a gene that is mutated and not working correctly together with the introduction of a new gene to the body which can help in fighting a disease. The primary method utilizes DNA which encodes a therapeutic and functional gene and replaces the gene which is mutated. Gene therapy shows a lot of potential in treating various diseases which include some viral infections, some kinds of cancer, and some disorders which are inherited. This technique is still under investigation to ensure its efficiency and safety and for the time being it is utilized in treating diseases with no other way to cure. The technique involves the introduction of nucleic acid polymers towards the cells of a patient like a drug to provide treatment for an illness. The polymers can be translated into proteins, affect the targeted gene expression or make a correction for the genes causing mutation. Gene therapy can be classified into four: germ line for enhancement, germ line for treating medical conditions, somatic for enhancements and for treating medical conditions.

Social and Ethical Issues associated with gene therapy

Autonomy which can be described as self-governance allows individuals to decide, reason and make a will. Legal, social and moral norms reveal the need to ensure autonomous agents are respected together with their choices. With this, it means that agents have right of self-governance without having control from the outside and such include the right to make an informed and independent decision concerning their wish of using gene therapy together with its consequences. Autonomy is also concerned with the right of people to have control over their destiny with without having to rely on genetic information while at the same time avoiding any interference in making crucial life decisions. Also concerned is the individual right of controlling utilization of genetic material provided for analysis per the specified need. Such includes the time when the genetic material and its information can be kept for future analysis either in registry file or DNA banks. However, in some circumstances autonomy can be overridden like in a case of preventing delicate harm to others as seen in mandatory screening for hypothyroidism and phenylketonuria in newborns (Ross, Saal, David, Anderson, & American Academy of Pediatrics. 2013). There exist those who argue that it is better treating diseases without causing any changes to the genes as they see it is risky given that the technology is recent and thus its long term effects are not known.

For legal issues concerned with autonomy, individuals need to be accorded the right of choosing to undertake gene therapy after being informed of the facts to consider in making their decisions which include the kind of their condition together with the prognosis it has, benefits and risks associated with the treatment and alternatives available. Individuals also need to have information and control subsequent utilization of any tissues taken from their bodies. Such can prevent conflicts of interest that may arise like in a case when an investigator may request an individual to undergo for a given test yet the researcher could be performing the same for commercial gain or own use. Even the sample will be utilized anonymously, the autonomy of the patient would have been compromised.

Privacy can be defined as a state of limited access. In gene therapy, privacy includes the right of making independent and informed decision concerning whom the details of a genome can be revealed to, and such can be spouses, insurers, researchers, other members of the family, researchers, employers, social agencies, institutions of learning among others. There are those who argue that privacy rights reflect property and personal rights like rights preventing one not to be overheard, looked at and be distressed. Privacy rights are justified to be crucial in intimate relationships for friendship and trust. They also allow people to have different types of relationships where different people are not equally accessible to everyone else.

Confidentiality can be described as a principle implying information to be sensitive and in such its access have to be controlled and be limited to those with authority to access the same. The condition of limited disclosure or nondisclosure can be protected by social, legal or moral principles. During medical care, access is granted to the body of a patient where it is observed, touched, listened to, physically invaded and even palpated. The body can be examined in whole or by using the only particular part where some parts like tissues can be taken for further investigations. Even though privacy is not taken care of when the bodies are accessed, confidentiality allows individuals to authorize and control further access of information gathered. A patient needs to be sure that physicians will maintain their confidentiality and that gives the reason why they allow them to have access while making diagnosis together with treatment entirely. Confidentiality rules are a requirement for the social welfare of the patient and for this reason they are not afraid of seeking treatments. There are those who argue that confidentiality rules can be infringed during gene therapy through breaches which are deliberate and also carelessness like in a case when the profession lack was taking adequate measures to protect confidential information. Arguments are given concerning physicians where they are obliged to infringe sensitive rules like when preventing an occurrence of a deadly harm.

Differentiating a medical condition and enhancement is not an easy task. Restriction of gene therapy to medical utilization has not been effective as a result of commercial and social pressure. Such enhancements include the application of gene therapy to cater for baldness. Other there are those who argue that human have the right to decide what is best for them, there are those who feel that people will not be comfortable with nature and thus will always work to cause changes to the same. This is mostly opposed by religions who believe that God had a reason for his creation and everything he created he saw was beautiful. There are those religions who argue that human was made to subdue the earth and can take the responsibility of changing whatever he/she feel like since the authority was given to them.

Germ line engineering allows children to be born without having carriers for recessive genes. With such children will not worry about reproductive challenges from their parents. There are those who argue that worries are and should be a part of the life of an individual and the best way to deal with them is by going to counseling.  They argue that worries should not justify the use of gene therapy and term it to be irresponsible. There are those who claim that for children cannot comprehend ramification that will result from gene therapy which goes against human autonomy (SOCIAL & Committee on Bioethics, 2013). There are those who also suggest that gene therapy will cause the world to be overpopulated.


In my opinion, I fully support gene therapy as it comes to save the life which is the most precious thing. Most of the concerns for gene therapy base themselves on misunderstandings together with lacking scientific information which makes them unjustified. Scientists have the role of improving scientific understanding to the public so that they can make their judgments based on knowledge rather than on prejudice. Even though it can be argued that gene therapy is risky, I would call for comprehension of its practice existing to provide a solution for challenges we face. By providing treatments for diseases, human suffering will be reduced, and thus the research will provide more help as compared to the harm caused.

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Maeder, M. L., & Gersbach, C. A. (2016). Genome-editing technologies for gene and cell therapy. Molecular Therapy, 24(3), 430-446.
Ramamoorth, M., & Narvekar, A. (2015). Non-viral vectors in gene therapy-an overview. Journal of clinical and diagnostic research: JCDR, 9(1), GE01.
Ross, L. F., Saal, H. M., David, K. L., Anderson, R. R., & American Academy of Pediatrics. (2013). Technical report: ethical and policy issues in genetic testing and screening of children. Genetics in Medicine, 15(3), 234-245.
SOCIAL, G., & Committee on Bioethics. (2013). Ethical and policy issues in genetic testing and screening of children. Pediatrics, peds-2012.

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