Gene Therapy



Gene Therapy is both a medical procedure and a condition that cures and treats diseases and gene related disorders. Over the years, gene therapy has been growing rapidly. The medical condition is a process of inserting genes into cells and tissues of a person for purposes of treating a disease that is believed to be inherited. Gene therapy concept is well compared to a transplant. However, the process of transplanting organs to human is slightly complex compared to gene therapy. As such, the process of transferring genes entails using various small molecules that are not visible with naked eyes. Such molecule cannot be seen even with the most powerful microscope. The aim of gene therapy is to supply mutant gene that is defective with another gene that works well (Naff, 2005). The technologies facilitating gene therapy is still growing, but has always been used with high probabilities of success. Although there are still puzzling questions surrounding the procedure. Therefore, to understand the process of gene therapy, it is important for one to fully understand issues to do with heredity. One of the foreseen applications of gene therapy is to treat patients with monogenic disorders bearing some form of recessive inheritance. Thus, the initial objective of the process tends to highlight the main features of the therapeutic modalities which have always been based on delivering additional copies of genes rather aiming at the correcting other faulty genes (Kelly, 2007). The latter objective would therefore need the development of technologies focusing on the homologous recombination of gene targeting according to the substitution of the tracts that carry mutations while the other one carries the normal sequence. The paper, therefore, discusses an overview of gene therapy, how it works, the safety of gene therapy, ethical issues behind gene therapy, and challenges of gene therapy.

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Overview of Gene Therapy

Gene therapy is an experimental health method that uses genes in treating or preventing a disease. The method allows doctors to treat a given genetic disorder than inserting genes into the cells of a patient rather than using some form of drugs or surgery. Scientific researchers have tested various approaches to gene therapies (Tomasoni & Benigni, 2004). Some of this approaches entail the  replacement of the mutated gene causing diseases with another copy of a gene (, 2017). Another process of facilitating gene therapy is through knocking out some form of a mutated gene that is not functioning well. The third process is introducing new genes into a body of an individual inorder to help fight certain diseases. Over the years the such gene therapy approaches have been a prominent option of treatment for various diseases. Some of this diseases include the inherited disorders, cancers, and other forms of viral infections. However, the technique has remained to be risky. For this reason, it is still subjected to various studies. This is important as it ensures that the method will still be effective and safe for the patient. The process of gene therapy is still under testing only for diseases that are yet to find their cure (Carmichael, 2014). Other approaches that have been used with gene therapy include swapping abnormal for a normal gene, repairing those abnormal genes, and changing the degree to which genes are either turned on or off. Although there are lots of hope for the process, gene therapy is still under experiment.

How it Works

Gene therapy works by introducing genetic materials to cells for purposes of compensating genes that are not normal. They are designed to introduce other genetic materials to cells in order to make beneficial proteins. Assuming that a mutated gene causes a protein to be either missing or faulty, the process of gene therapy is in a position to introduce normal copies of other genes in order to restore the normal functioning of the protein. The gene that is then inserted into the cell does not always function. Rather a carrier which is normally termed as the vector is then engineered genetically to deliver the gene. Some of the viruses are normally used as vectors since they tend to deliver new genes by infecting the cells. Other forms of viruses termed as the retroviruses tends to integrate the genetic material to the chromosomes found in the cells of humans. Other viruses like the adenoviruses then introduce the DNA molecules into the cell nucleus but the DNA molecule is then integrated into the chromosomes of the cell (Sadelain, 2004). The vector can either be injected or directly intervened to the specific tissues found in the body where it is then taken up cells. Alternatively, a sample of the cells from the patient is removed and then exposed to the vector within the laboratory setting. The cells that contain vectors are then taken back to the patients. In an event that the treatment is successful, the new genes will then be delivered with the help of a vector which will then make a protein that is rightly functioning.It is important for the scientific researchers to overcome various technical challenges before the process of gene therapy. For instance, it is important for researchers to find proper ways of delivering genes and targeting them to specific cells. They also need to make sure that new genes are properly controlled by bodies. Therefore, the premise of gene therapy has never been complex. If certain diseases are as a result of genetic mutations, then removing or even overcoming mutations needs to cure the disease. As such adding healthy versions of the malfunctioning genes tends to be anobviousand elegant mechanism of achieving this.

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How safe is Gene Therapy

Gene therapy has always been under study to find out if the process is safe when treating diseases. Current researchers are still evaluating the safety of the process. Future researchers will also test if gene therapy is an effective option for treating diseases. Various studies have already proved that this method tends to bring serious health risks like cancer, inflammation, and also toxicity. Since the treatment is still new, some other risks tend to be unpredictable. However, regulatory agencies, institutions, and medical researchers are working hard to make sure that the gene therapy treatment is safe (Verma, 2013).  For this reason, there are comprehensive federal laws, guidelines, and regulations that have helped people who take part in those clinical trials and research studies. The Food and Drug Administration regulated the products of gene therapy in the US and tends to oversee research in those areas. Some of the researchers who seek to test those approach in research studies need to come up with permission from the food and drug administration. The food and drug administration has the ability to reject such research studies that are being suspected to be unsafe to participants. The National Institute of Health has also been active in making sure that the safety of those gene therapy research is guaranteed. The institute offers guidelines for both the institutions and the investigators to abide when carrying out clinical trials with the gene therapies. According to the guideline, institutions that receive the National Institute of Health funding for this research need to be registered with the biotechnology activities board. The plan and protocols for every clinical trial are then subjected to review and analysis by the recombinant advisory committee (Philippidis, 2014). The purpose of the committee is to find out whether it will raise the ethical, medical, and safety issues that will warrant further discussion at the public meetings. It is important for the institutional review board to approve every gene therapy research studies before they can be implementedand carried out on patients. The institutional review board comprises of the committee of medical and scientific advisors and consumers who are charged with the responsibility of reviewing all the researchers within an institution. On the other hand, the Institutional Biosafety Committee comprises of relevant bodies that evaluate and reviews the potentiality of institutions. Multiple levels of oversight and evaluations make sure that the safety concerns are on top of the list when planningand carrying out the research on gene therapy.

Gene Therapy Ethical Issues

Since the process of gene therapy entails making changes to a body of an individual, there have been various issues to do with the ethical concerns of the process. Some of the ethical concerns that have surrounded the process include how bad or good use of the process can be differentiated, who is the custodian of deciding which traits and behaviors are normal and which of them are perceived to be disordered. Additionally, will the high cost associated with the process be only available to those who are financially stable. Could the vast practice of gene therapy make our society access those people who are different.Lastly, should individuals be told to go ahead with gene therapy for purposes of enhancing fundamental human rights like their intelligence, height, and also their athletic ability. Gene therapy researchers have continued focussing on treating people by targeting the therapy to cells of the body like the blood cells and bone marrow (Giacca, 2014). This form of gene therapy can never be transferred to one’s children. Gene therapy could target the germ cells, however, this would allow the genes that are inserted to be passed to generations. This form of approach is normally referred to as the germline gene therapy. The idea behind the germline gene has always been considered to be controversial. Although it might spare some of the future generations in a family from having thecertain genetic disorder, it tends to affect the development of thefetus in ways that are not expected or those that have long-term side implications that are yet to be known. This is because those people that are directed affected by the gene therapy are yet to be born. Therefore, they are not in a position to choose if they can have that treatment. As a result of this ethical concerns, the government of the united states has prohibited the federal funds to be used for clinical trials on the germline gene therapy.

Challenges facing Gene Therapy

There are various challenges facing gene therapy. First, it is quite difficult to deliver genes to the proper place and then switch it on. It is important that the new genes reach the targetted cell. This is because in an event that a gene is delivered to a wrong cell, then it could be inefficient and hence it could easily cause health implications for the patient. Even in a situation when the right cells have been targeted the gene needs to be rightfully turned on. The cells tend to obstruct the process by switching the genes off especially those that show some form of unusual activities. Another challenge is the process of avoiding some immune response. The function of the immune system is fighting the intruders that might try to come into the cell. Sometimesthis genes that are introduced into the cell by the gene therapy are potentially considered to be harmful intruders. Such responses are more likely to spark some form of immune response to the patient which might be harmful to them (Dalgleish, 1997). For this reason, scientists have the challenge of coming up with ways of delivering genes without noticing any form of theimmune system. This is normally done with the help of vectors that are less probable to trigger any immune system. Another challenges it to make sure that the new gene in no way will disrupt the functioning of other genes. This is because new genes that are introduced into the body by the process will integrate themselves to genomes of the patients and continue working for their entire lives. Thus, there is a risk that new gene will be linked to paths of other genes thereby disrupting their activities. This tends to have a damaging implication, for instance, if they interfere with animportant gene that involved in regulating the division of cells, then they could lead to cancer.

Summary/ Discussion

In summary, gene therapy is the process that occurs when the DNA molecules are introduced into a patient for purposes of treating a genetic disease. The new molecule normally has a functioning gene that would correct the adverse effects of a disease that causes mutation. The process uses sections of genes in treating or preventing diseases. The gene is selected carefully to correct the implication of the mutated gene that causes disease. Gene therapy was first developed 45 years ago and had recorded limited success to treat human diseases. Gene therapy is still subjected under research setting. The food and drug administration has approved a confined number of gene therapy products which are available for sale. Majority of clinical trials are under the process of testing gene therapy as one of the treatments for genetic conditions, cancer-related diseases, and also HIV (Philippidis, 2014). When one is interested in taking part in clinical trials, it is important for them to talk to their doctor or genetic practitioners on how they can participate. One can also look for other clinical trials on the internet.

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  1. Carmichael, L. (2014). Gene therapy. Minneapolis, MN: ABDO Publishing Company.
  2. Dalgleish, A. (1997). Why: Gene Therapy? Gene Therapy4(7), 629-630.
  3. Giacca, M. (2014). Gene therapy. [Place of publication not identified]: Springer.
  4. Kelly, E. (2007). Gene therapy. Westport: Greenwood Press.
  5. Naff, C. (2005). Gene therapy. Detroit: Thomson/Gale.
  6. Philippidis, A. (2014). Gene Therapy Briefs. Human Gene Therapy25(1), 12-16.
  7. Sadelain, M. (2004). Insertional oncogenesis in gene therapy: how much of a risk? Gene Therapy11(7), 569-573.
  8. Tomasoni, S., & Benigni, A. (2004). Gene Therapy: How to Target the Kidney. Promises and Pitfalls. Current Gene Therapy4(1), 115-122.
  9. Verma, I. (2013). Gene Therapy That Works. Science341(6148), 853-855.
  10. (2017). What is gene therapy? Retrieved 31 October 2017, from
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