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Gene Therapy: Risks and Benefits Research Paper

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Updated: Apr 18th, 2021


Gene therapy is the “insertion or removal of genes which can also be alternated within the cell or tissues of an organism for purposes of treating diseases” (Cross & Burmester). All over the world, “the technique is best known for the correction of defective genes so as to treat diseases; the most common procedural form of gene therapy involves the insertion of the functional gene in order to replace the mutated gene within an organism” (Cross & Burmester).

Over the recent past use of gene therapy has revolutionized the treatment approaches, and research on this subject is justified because of the great potential that this technique offers. Similarly, further research will also shed light on possible risk factors that are associated with this method of treatment. The purpose of this paper is to undertake a cost-benefit analysis of gene therapy as a treatment option.

Summary of the mechanisms of gene therapy

Germ-line gene therapy

Under normal circumstances, germ-line gene therapy procedures involve alternating and replacing all defective genes within the body of an organism (Walesgenepark.co.uk). In this case, functional genes are isolated and inserted into the interior lobes of the reproductive tissues or cells of the relevant organism. Considerably, this therapy operates on the basis that fresh and healthy genes get inoculated into the gametes of an organism in order to reduce the risks of later transfer of the defective genes to the next generation of the organism (Walesgenepark.co.uk).

Moreover, there is the involvement of complete alteration of the genetic makeup of early-stage blastomere so as to enhance changes of the genetic codes which can be passed on from generation to generation. Secondly, germ-line therapy also involves alteration of the gametes before fertilization since, after fertilization, the characteristics are passed on to the offspring’s (Walesgenepark.co.uk).

Somatic gene therapy

Generally, “somatic gene therapy involves the process of alteration of the genetic makeup of somatic cells (i.e., all body cells except sex cells) of an individual” (Rothchild, Laura & Lauren). Contrary to germ-line therapy, there is no transfer of these cellular changes to the next generation of the organism, simply because they are neither sex cells nor gametes which fuse during fertilization (Rothchild, Laura & Lauren). The main focus on the process involves changing the arrangements of the genetic codes of the specific cells using either an in-vitro or ex-vivo DNA delivery system. This technique in today’s life can be employed medically in treatments of a variety of diseases, including; hemophilia, muscular dystrophy, among many others; currently, it is used for cancer treatments (Rothchild et al.).

Discussion of paper and critique of the methods used

Telomerase inhibition strategies

In humans, it’s evident that the telomerase RNA abbreviated (hTR) plays an important role in anticancer therapy. This hTR can be used as an anticancer either individually or in combination with another human telomerase reverse transcriptase (hTERT) (Li, Li, Yao, Geng, Xie, Feng, Zhang, Kong, Xue, Cheng, Zhou & Xiao 4). Current findings indicate that when these two agents combine with the recombinant adenovirus, another nucleotide called small-interfering RNA (siRNA) is formed (Li et al. 4).

Furthermore, it has been established that the levels of telomerase activities together with mRNA, hTR are greatly reduced by the activities of the recombinant adenovirus resulting in inhibition of Xenograft tumor growth (Li et al. 4). This implies that siRNA, which is specifically expressed in recombinant adenoviruses, is the best tool to be used as an anticancer and also in the treatment of oral squamous cell carcinoma (OSCC) (Li et al. 4). The major advantage of this technique is that the anticancer effect on OSCC is virtually accomplished by cellular proliferation in addition to cellular apoptosis (inhibition of tumor angiogenesis) (Li et al. 4).

Monoclonal antibodies in target therapies of breast cancer

Globally, breast cancer is known to be a killer disease that largely affects females; the major causative agent in about 10% of world breast cancer cases is the mutation of the gene, which is inherited from any of the parents (Grammatikakis, Zervoudis & Kassanos 640). Furthermore, the most effective known therapeutic alternative diagnosis or treatment of breast cancer globally is the use of gene therapy. Some of the procedures used in treatment include molecular chemotherapy, antiangiogenic gene therapy, among many other therapies currently being used (Grammatikakis et al. 640).

bacteria-mediated anti-angiogenesis therapy

Most of the recent studies have revealed that there are some bacterial species that are capable of colonizing solid tumors. This inherited characteristic, however, can further be enhanced via genetic engineering, developing a natural anti-tumor activity that always enables the specific bacteria to transfer its therapeutic molecules directly into the target cells (Gardlik, Behuliak, Palffy, Celec & Li 7). There are few completed studies that have completely documented the anti-angiogenesis process, which is basically a bacterial mediated therapy for cancer (Gardlik et al. 7).

There are four recognized approaches that scientists use when utilizing the use of bacteria in cancer treatments; these include anticancer therapeutic-autofiction, DNA vaccination, transkingdom RNA interference, and alternative gene therapy (Gardlik et al. 7). Notable to mention is that the major primary goal of all these approaches is that they all focus on stimulation of angiogenesis suppression.

Argument and future experiments proposed

Based on the evidence of this paper, the discussion focuses on the argument of whether gene therapy is effective when the cost-benefit analysis is undertaken. Personally, I totally agree with the essay topic that gene therapy has more benefits than costs since it has been successful in the treatment of most chronic cancerous diseases. Gene therapy usually works by relying on immunotherapy and the use of vector organisms like viral particles to modify the genome of the host cell that triggers an immune response, which finally destroys the cancer cells present in the body (Cross & Burmester).

By using gene therapy, many cancerous diseases, i.e., lung, prostate, pancreatic cancers, have a higher chance of being completely treated. As such, gene therapy is now emerging to be the most common preferred treatment of choice because of its efficacy over other treatment methods (Cross & Burmester). The gene treatment also allows the use of a single vector or a combination of several vectors aimed at achieving optimal results (Cross & Burmester).

Currently, further research on gene therapy is still ongoing, and various clinical trials have so far been completed, which now has led to the evolution of vaccine productions (Cross & Burmester). The vaccines are promising to be the most effective technique since they only require autologous cells to have them manufactured, although this has many cost implications; this is one of the disadvantages of this technique. The second disadvantage is that very few hospitals globally are capable of having such vaccines manufactured because of the costly technology associated with the production of the vaccine. All these factors ultimately limit the availability of the vaccine as a viable treatment option.

A recent research study provides findings that show evidence of secondary gliomas stem cell, which originates from an astrocytic tumor that contains a genetic mutation that possesses the tumor suppressor gene recognized as p53 (Cross & Burmester). As part of the procedure, it was proposed in the experiments that in order to induce apoptosis of tumor cells, one must incorporate the use of an integrated suicide factor together with the adenovirus-mediated transfer of p53 (Cross & Burmester). Considerably, the main strength of this approach is supported by the fact that p53 mediate apoptosis follows two distinct and separate pathways reducing pathogenicity (Cross & Burmester).


Gene therapy technology has brought a great 21st-century revolution to the modern system of disease treatments, precisely when it comes to cancer treatments. It is remarkable to note that the development of anticancer treatments by modified immunotherapy and gene therapy, among others, has helped to cure many cases of cancers and saved many others from death. However, through gene therapy, many victims of cancer have attained a prolonged lifespan after being subjected to this treatment.

Scientifically, the principle behind gene therapy when it comes to cancer treatment is that a successful cancer treatment involving therapeutic modality always aims at real activation of death pathways within the cancer cells.

Therefore, there is no doubt that for cancerous diseases, the development of genetic engineering and specific cancer vaccines are proving to be the most effective treatment approaches. There are hope and confidence that in the near future, all obstacles encountered during the first generation cancerous and precancerous treatments will be eliminated by the development of second-generation modern therapeutic intervention as far as disease (cancer) treatments is concerned (Cross & Burmester).

Works Cited

Cross, Deanna., & James, Burmester. “.” Clinical Medicine and Research. 2006. Web.

Gardlik, R., Behuliak, M., Palffy, R., Celec, P., & Li, C. “Gene therapy for cancer: bacteria- mediated anti-angiogenesis therapy.” Clinigene Current Gene Therapy Weekly (2011): 7.

Grammatikakis, I., Zervoudis, S., & Kassanos, D. “Synopsis of new antiangiogenetic factors, mutation compensation agents, and monoclonal antibodies in target therapies of breast cancer.” Clinigene Current Gene Therapy Weekly (2011): 7.

Li, Y., Li, M., Yao, G., Geng, N., Xie, Y., Feng, Y., Zhang, P., et al. “Telomerase inhibition strategies by siRNAs against either hTR or hTERT in oral squamous cell carcinoma.” Clinigene Current Gene Therapy Weekly (2011): 4.

Rothchild, Allisa., Laura, Martin., & Lauren, Lubrano. “Gene Therapy and the Gametes.” Somatic Gene Therapy. 2010. Web.

Walesgenepark.co.uk. “What is Germline Gene Therapy.” Wales Gene Park. 2011. Web.

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IvyPanda. 2021. "Gene Therapy: Risks and Benefits." April 18, 2021. https://ivypanda.com/essays/gene-therapy-risks-and-benefits/.


IvyPanda. (2021) 'Gene Therapy: Risks and Benefits'. 18 April.

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