Gene Therapy : An Experimental Stage

Gene therapy has saved many lives but there also have been other cases where the treatment has not helped cure the patient’s genetic disorder and they have died. Take Jesse Gelsinger for an example. He suffered from an X linked genetic disorder of the liver so he was injected with an adenoviral vector carrying the corrected form of the gene (Couzin & Kaiser, 2005). This treatment was not a success and Jesse ended up dying four days later. He suffered from a massive immune response that was triggered by the use of the adenoviral vector that was injected in him to transport the correct gene into his cells, which caused him to have multiple organ failures and his brain to die.

Human gene therapy is a procedure that is currently being used to help fight diseases with no known cures. Gene testing removes a gene that causes a disease and replaces it with a healthy copy of that gene. By placing a healthy copy of the gene inside the patient’s body, that gene can help fight any diseases, such as different forms of cancer, inherited disorders, immune system discords or viral infections (AIDS). Once inside the cell, the genes produce what the patient lacks, it kills the diseased cells and it activates cells in the immune system.

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

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.

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.

Gene therapy has had many successes as well as setbacks. The success of gene therapy outweighs the setbacks Elizabeth would say. The first human to receive gene therapy treatment was a 4 year old girl with severe immune- deficiency disease; the disease was caused by a faulty gene that fails to produce a vital enzyme (Genetics, 23). Gene therapy

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

The simplicity and effectiveness of this technique suggests that gene therapy could be more widely incorporated in the management and prevention of diseases. Whilst researching the latest medical treatments being developed, I came to realise, many treatments will not be available on the NHS due to cost. This contradicted my childhood ideas of medicine; a profession of simply curing the ill no matter what. Reading ‘Doing Right’ by Philip Herbert gave me an insight into the core principles of medical ethics. Applying these principles to controversial issues such as the availability of treatments or the Charlie Gard case has helped me to better understand the basis of health economics and complexity of healthcare.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), but There are going to be many mistakes. What are we going to do with the mistakes?

Another facet of genetic engineering, gene therapy, has shown promise. This new process aims at removing genetic defects and certain personality traits (Begley, 61).

Many scientists and others believe that if gene therapy can be refined, it could be implemented to ultimately put an end to genetic disorders. The parents would have the children they want, and the children who had the natural disposition towards a genetic disorder could possibly become a normal child, whereas otherwise they would probably end up losing their life due to abortion. Those that support gene therapy view it as a win-win strategy:

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

Human beings are biological organisms that are all subject to disease. Disease is caused by infections which have various causes themselves. Infections may be caused by bacteria, in accidents or injuries, or even by abnormalities or idiosyncrasies from our genetic code. The immune system is responsible for fighting against infectious agents and protecting the body. However, for some a weakened immune system, especially a genetic predisposition to a weakened immune system, can leave individuals susceptible to dangerous diseases. That is where gene therapy comes in. Gene therapy is the replacement, or insertion, of an infected gene with a genetically altered and new healthy gene. The uses of gene therapy can open doors to many new cures for genetic diseases. It could be a cure for almost any abnormality as well. Therefore, with the help of gene therapy, human beings can benefit and even prolong their existence by preventing fatal diseases in future generations of families. Gene Therapy Gene therapy is the insertion of normal or genetically altered genes into cells usually to replace defective or diseased genes in the body. By using gene therapy, doctors can go to the source of the disorder or problem instead of prescribing a patient countless number of drugs (Hogarth, 2014). Gene therapy was designed to introduce new and corrected genes to compensate for any abnormal genes. If a mutated gene causes a protein to