Essay on Human Gene Therapy

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).

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

Although the treatment is costly and only available in clinical trials, gene therapy has treated some of the most known diseases, like Parkinson’s disease. People who are for gene therapy believe that it will change our world by getting rid of sickness. They think of a world without cancer and Parkinson’s disease. However, the people against gene therapy see a world with technology overtaking civilization. In their eyes, they think that gene therapy is wrong because of the side effects and the concept behind it. In addition, when thinking about gene therapy, the opposers conjecture that changing the genetic makeup of someone affected with genetic illness is wrong. I, on the other hand, believe that changing the genetic makeup of someone that is ill and suffering is exceptional because it is to their advantage. All in all, gene therapy is a heavily debated topic, but I believe that gene therapy can change our world in a positive

Imagine the possibility of eliminating serious genetic diseases from the world. Imagine the idea of treating, preventing or even curing diseases that are yet to be cured. Imagine the feeling of being given improved health and a prolonged lifespan. This can all be accomplished with the aide of genetic engineering. Human genetic engineering refers to the process of directly manipulating human DNA to produce wanted results. DNA is a simple but very complex chemical that has the power to change the world and has begun to do so already. Many opponents to gene therapy fail to realize that genetic engineering has great potential to become very important in the biomedical industry. Though controversy exists regarding the ethics of human genetic engineering, it can produce numerous benefits, which outweigh its disadvantages and side effects; therefore, scientists should be able to manipulate the human genome for the purpose of helping people with serious medical conditions.

Gene therapy is defined as the medical replacement of defective genes in living human cells; its aim is to replace the activity of a defective gene by activating a dormant gene which has a similar function (Wheale & McNally, 212). Under gene therapy comes the politically controversial Human Genome Project, a fifteen-year, $3 billion federally-funded biology program. The goal of the project is to isolate the defective gene on the chromosomes which comprise the human genome (Fletcher, 2). In this manner, the Human Genome Project may be able to rid the cancer-ridden genes from human DNA, thereby curing cancer permanently. The project has been the focus of much scientific and political controversy over the past few years for its possible ramifications are extensive to all of human existence.

Demolishing genetic diseases from the human race has been and always will be a key role in science. However, there comes a point in time when moral standards have interjected the betterment of cleansing genetic diseases. One of the many branches of science that has the complete capability to exterminate our destiny of any genetic diseases, gene therapy, is being silenced due to the curse of society’s moral standards. There is a very narrow line in what is not morally correct and ethical to standards of people; causing the advancement of procedures like gene therapy to be narrow. The only explanation to gene therapy awaits on what we, as a community, can bring about for the future generations, but this is

Scientists have projected there to be many benefits to gene therapy in the near future. For starters, gene therapy could cure diseases like cancer, aids, as well as thousands of genetic disorders (Will). Sara Goering, a philosopher concerned with biomedical ethics, argues throughout “Gene Therapies and the Pursuit of a Better Human” that germline therapy could mean disorders would be eliminated for good, leading to the relief of pain for many. It poses the possibility to eliminate human suffering at the root, and in turn, better humanity. Throughout society, people already focus on ‘enhancing’ their kids through vaccines and vitamins. Kids are out through surgeries because they are believed to be what is best. Goering points out that because of people’s willingness for treatments like this, there really are not many moral concerns prevalent (Goering 403-40). For many diseases, treatments that people already feel comfortable with will not work as effectively (“List”). Currently, there are no real cures for genetic disorders besides replacing defective

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 1990 the first gene therapy procedure gained approval and a four-year-old girl with SCIDs disease was finally able to fight off a simple cold. She is now able to live a normal life with the help of continued treatment of gene therapy. Although gene therapy is an innovative and ideally favorable procedure when it comes to treating diseases, cancer, or inherited disorders, it is still a delicate procedure and is continuously studied to insure it is not only effective but also and most importantly safe. In order to insure the safety of patients a gene therapy drug must get the approval of the FDA before becoming available, as every drug must do. Since gene therapy is still very experimental; there are only a few gene therapy drugs that have been approved by the FDA to treat patients. However, on August 30th, 2017 history was made when the FDA approved Kymriah, the first CAR-T cell therapy drug to be available in the United States. Kymriah is a one-time treatment for patients with B-cell acute lymphoblastic leukemia, it was developed through the research collaboration of Novartis and the University of Pennsylvania, and it is manufactured for each individual patient. Kymriah’s only drawback would be the price tag of $475,000 for a course of treatment, however Novartis plans to wave the fee for patients where Kymriah is not successful.

The purpose of this case study is to evaluate the political, economic and social impacts of Gene Therapy. Gene therapy is the “the insertion, removal, or alteration of a therapeutic or working gene copy to cure a disease or defect, or to slow the progression of a disease” [Ashish Verma, Anchal Singh – 2013].

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

“Although we are all members of a single species, we differ from one another in such visible traits as the color of our skin and the shape of our noses, and in biochemical factors such as our blood types and our susceptibility to certain diseases” (Sheridan College Institute of Technology and Advanced Learning, 2013, p. 34). To understand human physical development and evolution one has to understand biological anthropology as the focus on humans as biological organisms. Biological anthropologist conduct research, and form techniques of modern molecular biology to learn about human variation and how it relates to different environment humans lived in as well as their conditions.

Just as there are different types of people who look at one glass of water and describe it as half full or half empty, the public has many different views on the future of our society. Gene therapy is also a glass that can be viewed in different angles – different perspectives. Some say it has great potential to shape the ideals of our future, while others believe it signifies intolerance for disabilities, imperfections that supposedly deplete from a person’s interests, opportunities and welfare (quoted by Peter Singer, xviii). This global issue has brought people with different opinions in the open, arguing their views using history,

revolutionizing medicine and treating disease. It is believed that Gene Therapy holds the key to

Every living thing is the product of the genes that were passed down from ancestors. Genes make up everything we are. One gets their traits from their parents. Most people live full lives with relatively good health. However, some people inherit mutated genes or faulty genes. This could lead to genetic disorders that could be life threatening. Even today, many genetic disorders still remain incurable, leaving many people without hope. Genetic therapy could be their answer. It is through this research that the cure for genetic disorders can be found. Though some people believe it is unethical or immoral to alter genes, current therapeutics have not been able to save the lives of the patients with these diseases. Genetic therapy