Decoding Gene Therapy: Unlocking the Solution to Thousands of Genetic Diseases

Subject: Health Care
Type: Expository Essay
Pages: 5
Word count: 1330
Topics: Medicine, Health

Section One


Genes that are found on the chromosomes are the fundamental units of heredity. Gene therapy refers to the procedure of replacing defective genes in the tissues and cells with the healthy or functional ones. It can also be said to be to be the method of correcting the mutated genes that result in disease conditions. The correction or replacement of the defective genes aims to enable the body to make the functional protein and; hence, get rid of the source of the disease condition. In the past few years, various technological advances have been made, and this has enabled therapeutic trials in gene therapy to commence. The potential for this treatment is unlimited. Nonetheless, there have been numerous challenges that scientists globally have to deal with, including the question whether the benefits of gene therapy are more significant than the disadvantages.

Targets of gene therapy

Somatic gene therapy brings in good genes at the cellular level to treat a particular individual. On the other hand, Germ-line gene treatment introduces genes into the gametes or embryos to correct defects that could be transferred on to other generations (Bao et al., 2016). The potential extent of the gene therapy is massive. About 4,200 illnesses have been recognized as resulting from the defective genes, and innumerable other, which may be partly influenced by an individual’s genetic structure. Original studies have focused on the development of gene therapies for conditions whose genetic origins have been recognized and for other illnesses that can be treated or enhanced by substances the genes produce (Ginn et al., 2013). Gene therapy is targeted to be utilized in the treatment of various diseases such arthritis and cancers and also has been tested on individuals with HIV/Aids (Ginn et al., 2013). Gene therapy is aimed to supply the humans with a gene, which can rectify a biological failure that results in a disease in the future. Nevertheless, there are numerous complications and some different questions regarding the practicality of gene therapy.

Benefits and disadvantages of gene therapy

There are various social and ethical issues connected with gene therapy. An evaluation board has been formed that is called Recombinant Advisory Committee, to deal with the concerns linked with gene therapy.

The beneficial feature of the process is clear, as the gene therapy can eradicate diseases so that the next generations cannot inherit the malfunctioning genes. Gene therapy proves to be a potential medical solution that can end several genetic health conditions. Through the replacement of the faulty genes, hereditary diseases can be eradicated for good. Human beings have constantly been prone to various illnesses since when a cure for a certain disease is discovered, another illness appears (Ginn et al., 2013). People are continually attacked by new, more risky and vituperative kinds of pathogens. Even though a majority of such diseases can be cured medically, there exists no treatment for genetic conditions unless the malfunctioning cells are replaced with healthy ones, which is the purpose of gene therapy.

However, there are also numerous consequences of gene therapy. The first consequence is that gene therapy will put human fate in others’ hands allowing the individuals to produce future generations at will. Numerous people are at the view that gene therapy will be utilized to create superior race. Nonetheless, this is not the objective of the scientists.  The major plan of gene therapy is to eradicate genetic diseases (Bao et al., 2016).

Another concern connected with it is religion. Some individuals deem it sinful to change DNA. Nevertheless, in actuality, it is just a therapy employed to treat a particular genetic condition (Ginn et al., 2013). Besides, another major worry is the cost of the treatment that is very high and hence only favors the wealthy individuals since the poor cannot afford it. Therefore, it seems to ethically unfair or prejudiced. Nonetheless, the scientists are positive that the expense of gene therapy will be much reduced in the future. 

Moreover, the dangers of the procedure are also another source of concern regarding gene therapy. Because gene therapy is in its developmental phase, getting the exact location of the faulty gene and replacing it with a healthy one is absolutely a challenge (Bao et al., 2016). However, it is factual that the advance in technology and procedures, scientists will shortly be capable of accomplishing a huge success in the application of gene therapy. 


Currently, gene therapy is linked with both positives and negatives; therefore, the question that remains is if the gene therapy will, in future, eliminate the cons and have a spectacular effect on the future of genetics. Both the benefits and the disadvantages are significant, and hence the target of the scientists is to advance in the technology used to eliminate the negative sides related to the procedure for a genetic disease-free future.  

Section Two

Gene therapy is still under study to decide whether it can be employed to treat diseases. Existing studies are examining the safety of gene therapy; however, future research will analyze whether it is an efficient treatment alternative. Various studies have by now revealed that this approach can result in grave health threats, including toxicity, cancer as well as inflammation (Vogel & Motulsky, 2013). Since the procedures and technologies used are comparatively new, several dangers may be unpredictable; nonetheless, medical scientists, organizations, and regulatory bodies are working to make sure that gene therapy is as harmless as possible. 

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The benefits of gene therapy far overshadow the negatives. The technique is targeted to give individuals that are born with genetic disorders or who develop cancer the opportunity to lead a healthy life. Such an advantage surpasses any disadvantage that has emerged or presented against the process. It is ethical to ensure that all people in the society are leading pain-free lives without disturbances from diseases (Ginn et al., 2013). Ethics champions for human happiness and not suffering. Giving an individual a healthy life is sufficient for the people who oppose the technique to make them change their position. 

For instance, the germ-line gene therapy, which deals with the reproductive cells, may prove to be the only efficient way of dealing with some genetic diseases (Bao et al., 2016). Moreover, the germ-line therapy provides a right treatment, and not a just palliative and suggestive therapy. Through the prevention of transfer malfunctioning genes, the cost and dangers of somatic cell therapy and disease treatment for various generations is evaded. Moreover, this treatment will react to the reproductive health requirements of potential parents at threats of transmitting severe genetic disorders.

Therefore, the gene therapy will transform the performance of medicine. The responsibility of medics will be shifting in the years to come, and this will be because of gene therapy. Gene therapy approach can treat numerous health condition that has affected the society for many years and hence it with be ethical (Vogel & Motulsky, 2013). For example, the scientists have revealed the genes that lead to cystic fibrosis, which is a dangerous disease that reduces the life expectancy of the patients. Moreover, they have also discovered that various types of cancer are caused by genes in the cancer stem cells. Such diseases will be curable in the future because of gene therapy.


Gene therapy is an exciting advancement, which is currently just making the news. Soon, the technique will be a daily word utilized in the household. It will transform the field of medicine from what it is currently. As the biology experts in the field discover more genes and their roles, the potential of this approach is unlimited. The human genome is the blueprint of the human body. The key to human future is hidden in the genome. While scientists begin to comprehend this body design, people’s lives will be transformed forever. However, although there are some negative implications regarding the technique, improvement in technology and procedures will enhance the safety of the gene therapy for positive results. 

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  1. Bao, S., Lv, Z., Li, Y., Ma, J., Zhang, Y., Li, S. & Yang, F. (2016). Genetic Engineering: Pros versus Cons in Deciphering Disease Mechanism-ft. Science Insights. Doi: 10.15354/si.16.re203.
  2. Ginn, S. L., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013). Gene therapy clinical trials worldwide to 2012–an update. The journal of gene medicine, 15(2), 65-77.
  3. Vogel, F., & Motulsky, A. G. (2013). Vogel and Motulsky’s Human Genetics: Problems and Approaches. Springer Science & Business Media.
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