Treating Genetic Disorders with Gene Therapy Essay

Gene therapy is a very controversial topic that has been discussed in the last five years and is being heavily studied to help cure cancer. Gene therapy is a technique aimed at treating genetic disorders by introducing the correct form of the defective gene into the patient’s genome (Dunlop et al., 2010).

Gene therapy provides many benefits to the patients who undergo it. Biotechnologists believe that a genetic disease can be removed with this treatment. For example, on the U.S. National Library of Medicine website, it states, “Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or ‘knocking out,’ a mutated gene that

This is one of the major factors that lead to the absence in the gaining of therapeutic science. Although the lack in progress cannot be the only factor blasted for; some scientific trials have confirmed both anxious and fearful, advancing tighter on gene therapy restrictions. According to Hogarth, this hotly debatable topic of whether it is dangerous or not “playing God” has caused many religious, governmental, scientific and public figures to signify their own concerns in fierce debates (Hogarth para 9). Since the beginning of gene therapy, there has always been a constant battle over the guidance of its future of whether gene therapy will transform into a therapeutic science or lead to unpleasant consequences as the eugenics did in the 20th century. The ethics of gene therapy have been established and based on, not only beliefs, but also the disconnection and advantages achieved in the trials, which has lead to the breakthrough of the future of gene therapy. The downfall and advancement of gene therapy are the hereditary products of research that have had a major influential impact on its ethical features. When gene therapy was a novice, it gained an immense amount of popularity when scientific research evolved towards identifying genes that induce certain diseases (Hunt, 2006).

Fifty years after the idea of gene therapy was first proposed, gene therapy has become a possible treatment for a couple different diseases. Before this treatment was approved, some serious unfavorable effects were found in clinical trials. However, these effects fueled more basic research in order to improve, in efficiency and safety. Gene therapy has been used for patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer.

In the future, medical professionals hope to be able to treat or even eradicate a disorder by inserting a new gene into the cell via drug or surgery that will combat or even fix the mutation in the DNA that causes that disease. This therapy is still in the very early stages of experiments, and the ethical questions surrounding it prevents much progress from being made at all. There are many potential health complications surrounding this type of approach: scientists still do not completely understand how genes work and if they can be replaced. CRISPR will be a major tool used in gene therapy. CRISPR, which stands for , is a unique technology that lets geneticist and medical researches edit part of the genome by pinpointing a certain area of DNA and adding DNA, removing DNA, or altering the DNA sequence (Your

Just in case you are a little shaky on the topic of gene therapy I will briefly explain. Gene therapy is a type of treatment used

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

There are two types of gene therapy; germline gene and somatic gene therapy. Germline gene therapy is considered to be a safer option for humans. This method makes changes to the gamete cells that are used in the reproductive process thus, the functional genes need to be inserted into the chromosomes. Somatic gene therapy is when genes are transferred to body cells by inserting vectors into a person’s body. There are three things that gene therapy can do; replace a mutated gene that causes a disease with a healthy gene, deactivate a mutated gene that is not able to function properly, or introduce a new gene into the body to prevent diseases. There has been a clinical trial in the past where over 3000 people were treated with gene therapy and the results were good however, this treatment method has serious health

Akudo Ejelonu writes, “The medical industry is building upon the knowledge, resources, and technologies emanating from the Human Genome Project to further understanding of genetic contributions to human health....Genetics is playing an increasingly important role in the diagnosis, monitoring, and treatment of diseases” (What is the Human Genome Project-Why Is It Important To Society par.10). According to this evidence, the Human Genome Project has opened doors for scientists to better understand the use of gene therapy for humans. Genetics is now playing an important role in curing, monitoring, and diagnosing diseases that can make someone very sick. With gene therapy it can save lives and help cure the world of its deadly diseases. “Genetics Home Reference” states, “ Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS” (Gene Therapy Experimental Techniques, Safety, Ethics, and Availability par.15). The “Genetics Home Reference” is insisting, gene therapy is on its way to becoming a treatment for anything and everything. It is on its way to becoming a full time treatment for diseases that will later be cured because of frequent gene therapy sessions with sick patients. All of the hundreds of research that has been done on this subject is just a grasps an understanding of how it all works and how it changes the world in a positive way. Larissa Hirsch states on page three, “Scientists hope that the human genome mapping will help lead to cures for many diseases and that successful clinical trials will create new opportunities”(Gene Therapy and Children par.4). Larissa Hirsch is stating that the Human Genome Project plays a big role in gene therapy. The Human Genome Project is opening doors for scientists that have successfully performed medical trials when it comes to treating

Gene therapy is a rapidly developing technology first used in 1990.1 It involves the use of modified DNA inserted into an organism with a missing or mutated sequence. There are two methods of delivering this correction into the patient’s cells. In the viral method, a virus containing the vector with the corrected form of the gene, infects the cell naturally. Adenoviruses, adeno-associated viruses, lentiviruses, and retroviruses can all be used. They each utilize a different method of delivery and form of DNA. Adeno-associated viruses and lentiviruses are the easiest forms to construct.2 The corrected DNA faces many obstacles to gene expression and ultimately protein production. The viruses must target and enter the correct cell, avoid

Gene therapy works by adding a good copy of a defective gene to the cells in need (“What is”). Gene therapy is different than traditional treatments because it goes right to the source of the problem, rather than just treating the symptoms (“What is”). Although gene therapy is a possible treatment for many medical conditions, conditions affecting many genes is not the best candidate for gene therapy (“What is”). Inserting the gene into a cell can be an extremely difficult process. To insert the gene into a cell, a virus is attached to a vector (Vachani). This vector is used as a carrier and is inserted into the body with the new gene attached. The gene then goes into the cell’s nucleus and becomes attached (Vachani). There are multiple ways to give gene therapy to people and researchers can find the most effective way. Since gene therapy is still new and being experimented with, the side effects are still unknown (Vachani). After the vector is inserted, the body may react and cause a fever, chills, nausea, and a blood pressure drop (Vachani). These side effects are usually gone after a day or two (Vachani). Although it has not happened yet, researchers are worried that the new genes could start to affect the healthy cells (Vachani). Depending on which cells it affects it could

Gene Therapy is a complicated and very scientific practice that takes place on a genetic level, hence the name. As mentioned in “What is Gene Therapy,” it was originally created in 1972 and at first had very limited success. There are two main ways to go about it, both distinct opposites, as explained in “List of Pros and Cons of Gene Therapy.” The most common, and currently only legal form of gene therapy is somatic cell therapy. This kind of treatment targets the body’s somatic cells which are any cells besides reproductive cells. Because of this, the treatment is only corrective for the specific patient and cannot be inherited by offspring. The other main kind of gene therapy is germline therapy which is currently banned in the United

Moreover, Positive aspects of gene therapy are apparent as it can give many peoples a new life. It can reduce the stress on future parents as they do not have to think or worry about having a child with disabilities they can have a normal child because gene therapy can wipe out genetic disease before they can begin and eliminate suffering for future generations; it is also good technique for diseases that are not searched yet because each of us caries about half a dozen defective genes, however we remain ignorant to this fact unless we are among the millions of people who have a genetic disorder; about one in ten people has or will develop some time later in life an inherited genetic abnormality. It and can also silence a gene in the case

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. ("Gene Therapy," n.d.). In the United States, gene therapy is only available in a research setting.

The genes we inherit from our parents virtually affect every human disease. The argument for gene therapy lies in the understanding of the genetic basis of human disease. There are an amazing 100,000 individual genes in the human genome. At the present time there is an international movement known as the Human Genome Project. The project will eventually provide an understanding of how each gene plays a role in human lives. The project