Introduction
Gene therapy refers to the “deliberate introduction of genes into an organism” (Lerner & Lerner, 2008).According to Lerner and Lerner (2008), gene therapy helps to correct a genetic defect. In case of a patient suffering from a genetic disease, gene therapy can also be used to ease symptoms (Lerner & Lerner, 2008). In other words, gene therapy entails the use of corrective genetic engineering to correct defective genes.
Biological Basis
Gene therapy is an offshoot of molecular biology. The cell is the basic building block of human life. The cell nucleus is made up of threadlike structures called chromosomes whose basic content is deoxyribonucleic acid (DNA). DNA acts as a carrier of genes. Genetic diseases like sickle-cell anemia are as a result of defective genes. Scientific studies show that individuals with distorted genetic sequence are more likely to be diagnosed with cancer and schizophrenia compared to individuals with proper genetic sequence. In light of this, gene therapy aims to replace dysfunctional or missing protein through the introduction of corrected genes (Lerner & Lerner, 2008).
How Does Gene Therapy Work?
According to the U.S. National Library of Medicine (2014), gene therapy works to correct a defective gene by introducing the corrected genetic materials. Also, gene mutation can lead to loss of protein function and if this happens, gene therapy helps to restore this. Modified viruses (through genetic engineering) act as vectors or vehicles that deliver the gene into the cell.
What does gene therapy accomplish?
Germline gene therapy is among the two widely used types of gene therapy. It entails modifying germ cells genetically. The modified germ cells carry the modification to future generations (Moreland & Rae, 2009). In the other common form of gene therapy (Somatic gene therapy), the modified gene cells are only corrected in the patient and the next generation does not get to inherit them.
Accomplishments
There are a number of examples where gene therapy trials have proved to be successful. A good example is the trial of gene therapy on SCID (severe combined immune deficiency). Follow up studies have revealed normal immune function in patients who underwent the trial. Gene therapy has also been involved in the treatment of various forms of cancer like lung cancer, ovarian cancer, and breast cancer, among others. Trials of gene therapy have also been conducted to treat patients who are genetically predisposed to the development of Alzheimer’s disease, asthma, and breast cancer before the clinical manifestations of these diseases start (Friedmann, 2007).
Social and Ethical Implications
Gene therapy is, for the most part, mainly experimental and as such it poses significant safety concerns. A number of gene therapy applications are aimed at bringing about the “stable modification of the genetic characteristics of an individual” (Giacca, 2010, p. 283). The idea that such modification is ethically sound has led to a heated debate. Gene therapy is largely acceptable as long as it is used to ensure the survival of an individual or to improve his/her health. However, the exploitation of gene therapy for aesthetic, intellectual and/or physical appearance has raised ethical concerns.
The exertion of toxicity to cells by viral vectors is another area of concern. For example the Adenoviral vectors are immunogenic and inflammatory by nature and as such, there is the fear that this could lead to cell transformation. Another issue of concern is the possibility that certain gene delivery procedures could in fact be toxic. For example, during angioplasty where transfer of genes occurs on the arterial wall, the procedure demands that the catheters used should partially block the flow of blood so that the therapeutic nucleic acids can be injected (Giacca, 2010).
Considering that gene therapy does not come cheaply, a number of political commentators have raised concerns that this could give the rich an undue advantage over the poor. In addition, ethicists have expressed concerns about a possible misuses of this technology for other purposes, other than correcting defective genes. For example, there are fears that some scientists might exploit the technology to enhance human intelligence. Since gene therapy entails a modification of the basic human structure, religious leaders are concerned that this amounts to “playing God” and are hence opposed to it.
Risks and benefits
Moreland and Rae (2009) have examined various risks associated with gene therapy application. First, Moreland and Rae (2009) state that in a situation where there is a wrongful insertion of the working gene, this poses a great danger to the patient. Another potential risk could be that the deactivated virus might actually prove to be contagious (Moreland & Rae, 2009). Concerns have also been raised about the possibility of gene therapy causing an inflammation to the patient’s immune system as a response to the new genes inserted. On the other hand, gene therapy is associated with a number of benefits. First, gene therapy has demonstrated huge potential to cure, ameliorate, or prevent diseases with inadequate or no treatment. For someone born with a genetic disease, gene therapy gives him/her a chance to live a normal life.
Personal viewpoint
With gene therapy, there is hope for finding a cure for genetic diseases such as cancer. On the other hand, concerns have been raised about the ethics of using this technology. Nonetheless, somatic cell gene therapy holds a lot of potential in finding a cure to most genetic diseases. For effective realization of the benefits of gene therapy, the government in collaboration with the scientific community and various public associations should develop useful guidelines to govern the use of this technology. People of all walks of life should have access to gene therapy, and not just the rich. Funding of trials in gene therapy should also involve the government and the private sector. In addition, scientists and government regulators need to create awareness on the benefits and possible risk factors of gene therapy application.
Conclusion
Gene therapy involves replacing malfunctioning or missing genes to a patient’s cells with new genes. This is normally accomplished using a virus as a vector or vehicle to transport the new genes to the patient’s cells. Gene therapy is an outgrowth of genetics. The cell is the basic structure of a multicellular organism and each cell has its own definite role. The cell’s nucleus carries pairs of chromosomes which in turn carry genes. In case of any alterations on genes, this is likely to cause genetic diseases like hemophilia or sickle-cell anemia. Gene therapy therefore involves manipulating the gene’s structure through genetic engineering in order to correct such genetic defects.
Since gene therapy entails alteration in the body’s genetic makeup, this has raised a lot of unique ethical concerns. So far, only somatic cell gene therapy has been conducted in different clinical trials. However, there is a concern among ethicists that improvements in the clinical trials on germ line gene therapy could lead to its exploitation to enhance aesthetic, intellectual and physical appearance. Therefore, there is need for players in the fields of government, medicine, religion, biology, and politics to formulate ethical guidelines to govern research in gene therapy.
Reference List
Friedmann, T. (2007). A Decade of Accomplishments: Gene Therapy and the ASGT. Molecular Therapy, 15(9), 1576-1578.
Giacca, M. (2010). Ethical and Social Problems of Gene Therapy. New York: Lerner, K. L., & Lerner, B. W. (2008). Gene therapy: The Gale Encyclopedia of Science, 4th ed. Detroit: Gale.
Moreland, J. P., & Rae, S. B. (2009). Body & Soul: Human Nature & the Crisis in Ethics.Downers Grove, IL: Intervarsity Press.
U.S. National Library of Medicine. (2014). How does gene therapy work? Web.