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).

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.

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

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

Scientists have been researching genes and DNA throughout the 20th century. In 1990, the first successful gene therapy experiment occurred to a young girl with Severe Combined Immunodeficiency (SCID). This, although did not cure her, helped her continue with a relatively normal life. Again in 1999, an 18 year old man, with a liver disease, was in a gene therapy experiment. During this he had a reaction to the adenovirus and died of organ failure. In 2000, the Senate began discussing the topic of gene therapy research. In France, in 2002, three children with SCID were part of another gene therapy trial. They all were later diagnosed with leukemia, and one of them died. In 2005 the FDA declined to speak about the continuation of gene therapy trials.

Li, and Huang discuss the controversial topic of gene therapy by first providing a factual summary of gene therapy. Their introduction goes into the specifics of the types of vectors (carriers that deliver a gene to target cells) used to transfer genes in order to aide damaged proteins. Afterwards, they transition into the detailed of process of gene therapy. They start this off with naked DNA and state it is the simplest approach to nonviral transfers and the least toxic method. However, despite being the least toxic and a nonviral transfer it does not have as great of an effect as Cationic lipids. Cationic lipids have been used in various experiments but are known to be safe only when administered in small doses. Furthermore they go into

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.

There have been multiple setbacks in this technology. Some immediate side effects is that the body thinks that the virus inserting the healthy gene is a virus and tries to fight it. A severe response to the virus could lead to the death of the individual. Another major side effect in the long run is the development of other diseases. There have been multiple reports of operations that have gone successful, but in a few years that patient developing diseases like leukemia and most of the patients died. Right now there are probably more negative effects to gene therapy and more research would need to be developed to ensure that the likelihood of death is not so high. More research and operations on animals would have to be done to ensure it is safe on

For instance, according to the Therapeutic Goods Act 1998 (Cwlth), all human clinical trials, including Gene therapy, “must operate under one of the following schemes; the Clinical Trail Notification (CTN) or the Clinical Trial Exemption (CTX). All human clinical trials must also be overseen and regulated by the Therapeutic Goods Administration (TGA)” [Dan Bui, 2017] in order to ensure the safety and security of consumers.

With our current society rapidly advancing in both the technological and medicinal world, many new treatments have been born that can be used to treat genetic conditions that regular antibiotics and surgeries simply have no positive or progressive effect. Gene therapy is when a newly developed gene is transferred, or introduced, to an already existing gene, it is the attempt to treat one’s genetic disorder at the molecular level and could significantly improve the human population and provides hope to people with disabilities. Gene therapy goes straight to the source and alleviates symptoms of the disorder. The gene receiving this new development is in some shape or form, mutated, or defective. If the gene therapy is a

I read an article on an online news source that the FDA has authorized the use of gene therapy to try to cure a form of leukemia called B-cell acute lymphoblastic leukemia, or ALL. This is the first FDA approved gene therapy in the United States. ALL can affect children and adults. The trial is targeted to children and adults under the age of twenty-five who haven't responded to previous treatment, and who havn’t had a relapse. The scientists are able to alter a patient’s T-cells by adding a new gene to the cells. This new gene directs the T-cells to target cancer cells. This is a revolutionary trial that will open many doors in the scientific community in the future. More and more experiments with genes will occur. This trial of gene therapy affects body cells, specifically white blood cells.

At a simplistic level, gene therapy is the use of one’s own cells to provide a therapeutic effect. In the majority

Gene therapy also cured several kinds of cancer, such as suicide gene therapy, oncolytic virotherapy, and anti-angiogenesis and therapeutic gene vaccines (American Society of Gene & Cell Therapy, 2015). According to American Society of Gene & Cell Therapy (2015), “two-thirds of all gene therapy trials are for cancer and many of these are entering the advanced stage, including a Phase III trial of Ad.p53 for head and neck cancer and two different Phase III gene vaccine trials for

I volunteer at the Houston Medical Center whenever I go back home and a gene therapy program has been implemented in M.D. Anderson for cancer patients. It’s been at the forefront fighting rare genetic disorders that affect children. Their successful trials excel every step in the gene therapy process. While doing research, I also stumbled upon Dana-Farber’s cancer and blood disorder center where another gene therapy program has also been implemented [2]. Through their research capabilities, they research on basic studies on the causes of genetic diseases, gene/target identification, vector selection and development, preclinical validation and development, monitoring the efficacy and safety of genetic modification, development of new strategies for genetic engineering, and identification of novel clinical applications [2]. They work with multiple partners and conduct patient cell transduction and prepare gene corrected patient cells for infusion. I believe that their gene therapy research is truly effective and everyone should be completely for gene therapy research. In fact, just this past year in August, the Food and Drug

seems to hold the key to make this a reality. Gene Therapy is still in its early stages and is still