Gene Therapy: Two Treatment Options to Boost Immunity - Essay Sample

Introduction

Gene therapy involves the removal of DNA that does not work as intended and introducing a new one, which can aid in boosting one's immune system (Dunbar et al., 2018). It is vital to note that there are two kinds of gene therapy treatment options, including somatic and germline techniques. Somatic involves the transmission of a gene to a body cell that does not facilitate reproduction. It indicates that the strategy does not allow the transfer of gene therapy's effects from a patient to one's offspring. The germline strategy entails the placing of a piece of DNA on body areas that enable the production of sperms or eggs within a human body. It allows the transfer of the medical alterations from one generation to another.

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Importance of Gene Therapy

The significance of gene therapy is that it involves the manipulation of genes within body cells to treat or manage a disorder. Genes have DNA that regulates the running of a human body's functions. It indicates that when genes have a defect, it can promote the development of a disease. Experts prefer to employ the concept of gene therapy since it promotes reproduction, elimination of disorders, and a sense of flexibility during innovations (Haggerty & Dinsdale, 2017).

Basic Process of Gene Therapy

There are three stages of conducting gene manipulation (Naso et al., 2017). The first phase of doing gene therapy is to evaluate the cause of an infection. The second step involves creating a duplicate DNA which matches the defect gene to solve the health issue. The last stage is to create a system that can deliver the new gene into a patient's human body. The method of transferring DNA involves the use of a vector to store the substitute gene. There are two types of vectors, which include viral and non-viral. Viral vectors such as retrovirus are the most common vectors since they can carry normal DNA. The hereditary material contains instructions that initiate the manufacturing of protein, which enables the management of various types of diseases among patients, for instance, infectious illnesses. Gene therapy employs several techniques, including augmentation, inhibition, and cell destruction (Nyamay' Antu et al., 2019). Augmentation and inhibition are in-vitro gene manipulation techniques since they do not entail the removal of the defect. But the destruction method can also get referred to as ex-vivo gene therapy since it prompts the eradication of genes from the human body. Gene augmentation involves the eradication of mutation caused disorders in a body cell. It can apply in the treatment of diseases such as cystic fibrosis. It consists of adding DNA, which contains a function that the other gene lacks within the cell. The figure below summarizes the process of gene augmentation within a human cell;

Inhibition strategy is useful for illnesses with unsuitable gene activities such as cancer, infectious, and genetically transmitted diseases. The method undermines the inappropriate DNA's impact on the body cells. It eases the medical experts' ability to impede the development of diseases within their patients. For example, the inhibition technique is a suitable method for eliminating cancerous cells since it discourages oncogene activity (Dunbar et al., 2018). Destruction of abnormal cells is a suitable technique for cancer patients since it eradicates the spread of the illness within a human body. There are two methods of employing the process, which include the use of a gene that destroys a cell or allowing the immune system to attack the flawed DNA (Haggerty & Dinsdale, 2017). Medical specialists can introduce a DNA with a gene that contains a toxic substance which can ensure the elimination of a disease. Scientists can also use a gene that makes the diseased cells to become a target for the white blood cells to destroy hence ensuring removal (Naso et al., 2017). The figure below illustrates the entire process of gene manipulation;

Genetic Toxicology

Genetic toxicology is a branch that analyzes the impact of chemical and physical factors on a living organism's DNA and genetic activities (Roman-Rodriguez et al., 2016). There are various ways of conducting genetic toxicology. An example is analyzing the interaction between different causative agents and DNA, evaluating genetic material damage, and using gene alterations. Gene toxicology involves the analysis of measures that cannot get transmitted from one generation to another. For example, DNA strand breaks and unscheduled DNA synthesis (Maeder & Gersbach, 2016).

Mechanisms of Gene Damage

Various factors contribute to the damage of DNA within a human body, such as bases, radiation, and hydrocarbons (Roman-Rodriguez et al., 2016). There are several damages related to the DNA, which include oxidative and hydrolytic. Oxidizing various bases within genes can encourage the development of damage that can promote brain-related infections. The impact of the alkalis can get accelerated when exposed to chemical substances such as carcinogens. Hydrolytic loss entails the eradication of bases, which can hinder transcription of a person's genome. Various factors lead to hydrolytic gene damage, which includes biochemical processes and excessive oxygen species. The presence of radiations, such as ultraviolet, can undermine genes since it encourages the development of strand breaks (Roman-Rodriguez et al., 2016). It is dangerous since the cuts in DNA strands can affect genetic expression. Damage caused due to Ultraviolet rays can promote the creation of pyrimidine dimers, which challenges the process of replication hence change a person's genetic composition. It facilitates genetic mutation, which makes people become skin cancer patients. Individuals can experience genetic damage after getting exposed to polycyclic hydrocarbons, which are air pollutants related to smoke and oil. Medical officers employ the comet assay tool to evaluate whether an individual has any kind of DNA damage.

Goals: Better Vectors

In the current world, few vectors can get employed when conducting gene therapy. For example, clinical trials rely on the AAV-2 vector when evaluating the impact of the technique on various disorders (Maeder & Gersbach, 2016). It is because the vector is non-toxic hence cannot cause harm within a human body. There should be a wide range of vectors with high efficiency that will enhance the aspect of automation when introduced into a body cell. It illustrates that the top precision vectors will make it possible for medical specialists to deal with complex disorders due to active targeting. Researchers have the goal of increasing the size of vectors to maximize the number of genes that they can carry into a human genome. It will eradicate the current challenge of dealing with small-sized vectors such as lentivirus, which cannot apply when transferring a transgene.

Future of Gene Therapy

Experts are still conducting evaluations to determine gene therapy (Shapiro et al., 2018). Scientists may create delivery techniques that will directly introduce new genes to patients' body cells without the need for vectors (Simonelli et al., 2010). In the next years, researchers will develop strategies that will ensure the introduction of gene therapy to treat various diseases such as cancer, cardinal related disorders, and even HIV/AIDS (Shapiro et al., 2018). In the United States of America, the government will approve the use of the technique in eradicating various ailments such as Leber congenital amaurosis. There is a possibility that there will be the use of DNA manipulation among human embryos, which will eliminate the presence of genetic disorders before further development. Investigators will continue to develop the genetic testing approach by detecting the specific region of gene mutation (Shapiro et al., 2018). It will ensure that medical specialists enforce techniques that ensure precision during the DNA therapy process.

Gene Therapy's Safety

Experts indicate that gene therapy is an approach that poses health risks to its users, such as adverse cases of cancer inflammation (Maeder & Gersbach, 2016). Due to its unsafety, agencies established guidelines and regulations to ensure the analysts' protection when conducting clinical trials. It is the duty of the Food and Drug Administration (FDA) agency in the U.S to reject gene therapy examinations that can endanger the lives of the subjects (Verhoeyen et al., 2016). The National Institutes of Health (NIH) organization has rules that learning institutions and health facilities should follow when evaluating the gene therapy process (Roman-Rodriguez et al., 2016). It is essential for facilities that receive NIH's financial support to provide the agency with their plan for review, which can get rejected; it raises any ethical or safety concerns. An institutional review board (IRB) needs to validate a clinical trial before scientists get permission to conduct the examination (Verhoeyen et al., 2016). IRB is a group of scientists and analysts that have the responsibility of evaluating studies within an institution. The committee has to rule out researchers that have unsafety issues since it can increase the risk of health effects.

Clinical Trials

DNA Therapy on Eye Infection

Leber congenital amaurosis is an example of an eye infection that makes it difficult for individuals to have a high visual capability, enhanced light sensitivity, and lack of eye movements (Simonelli et al., 2010). It is a retinal disease that can attack individuals from an early age and can get transmitted from one generation to another. Simonelli et al. (2010) researched to evaluate the accuracy and safety of using DNA therapy in the treatment of Leber congenital amaurosis disorder. The evaluation indicated that the introduction of adeno-associated viral vectors helps in the eradication of the RPE65 gene, which undergoes mutation hence causes the occurrence of the eye-related disease (Simonelli et al., 2010). The collected information illustrated that the gene augmentation concept promotes the management of the retinal infection.

Gene Therapy on Organophosphate Intoxication

Cowan et al. (2001) conducted a clinical trial to examine the impact of gene therapy in enforcing the protection of the central and peripheral nervous body organs from toxins within a human being. It is the work of enzymes such as paraoxonase to act as a protective barrier to prevent injuries. A person's genome determines the level of the enzyme concentrations in the liver and bloodstream. The research focused on assessing the impact of organophosphate before and after using gene manipulation. The clinical trial relied on mice as the subjects of the evaluation. The findings showed that gene therapy aids in boosting the enzymatic levels since the research involved the introduction of a destructive vector before and after in toxification (Cowan et al., 2001).

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