I Will Choose Gene Therapy

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.

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.

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.

This study is about the administration of treatment using advanced methods made available over the past 20 years. It will attempt to explore the three most significant include Gene therapy, Stem cell transplant and health screening, commencing with meanings, approaches; uses and case studies of each type of techniques.

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.

(9) in the future, gene therapy may be approved, not to promote genetic advantage or privilege in offspring, but rather to remediate genetically or otherwise physically inherited genetic diseases and pathological conditions.

A new form of medicine with the potential to save and improve millions of live is gene therapy, or gene replacement therapy. Gene therapy is the use of DNA as a pharmaceutical agent used to treat or cure diseases or disorders caused by defective or missing genes. There are three methods of gene therapy: replacing a mutated gene with a healthy copy of the same gene, inactivating a gene that is not functioning properly, or introducing a completely new gene to help the patient fight a disease. Gene therapy is a promising treatment option, however, the technique has yet to be perfected and is still dangerous and experimental.

gene therapy is being used to develop recombinant cancer vaccines. However, they do not work

The FDA had, in August of 2017, approved the first gene therapy product available in the United States [7]. They approved Kymriah, a CAR T cell therapy, for children and some adults with acute lymphoblastic leukemia. Kymriah involves collecting a patient’s T cell, a part of their immune system, modifying the cell via adding a protein which can help the T cell destroy Leukemia cells, and adding the modified T-cells back inside the patient. It is, in theory, similar in design to the gene therapy which DeSilva had participated in. There are currently 16 gene therapy trials approved by the FDA [10]. The FDA is also take actions to speed the approval of gene therapy [11], as it released a regenerative policy framework on November on 2017 which gives companies working the gene therapy space guidance on what regulatory actions they must take [13]. While gene therapy certainly has regulatory and ethical challenges, it is becoming more and more common. As stated by FDA Commissioner Scott Gottlieb, “This [gene therapy] is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine”

The process of gene therapy is the introduction of a “good,” or normal, gene into a person who is affected by a “bad,” or mutated, gene. The doctor is manipulating the genes in the child to try and treat whatever disease the individual has. There are 2 types of gene therapy, somatic and germline, and in both ways there is no way to get away from the disease. In somatic gene therapy, the doctor injects the normal gene into the patient’s targeted cells to try and treat them, but the new gene does not get passed on to the offspring of the patient. In germline gene

We carry about a thousand genes in us. Most of the genes we inherit come from our parents. Hundreds of genes can contribute to common conditions like breast cancer, Alzheimer’s disease, colon cancer, and rare disorders like cystic fibrosis and muscular dystrophy (Harvard Women’s Health Watch, 1998). Gene therapy is known to treat genetic diseases and other health problems. Figure 1 illustrates how gene therapy works. Genes are introduced into a human’s body by replacing the bad cell with a new cell. In Katherine Hunt’s article, (Gene Therapy), it explains the history of gene therapy that took place between 1970’s through 1990’s. In the 1970’s, scientists came up with “gene surgery,” to treat inherit diseases that are caused by bad genes. In

George Santayana once said, "Those who cannot remember the past are condemned to repeat it," applies equally to technology issues such as gene therapy today. Therefore, I would like to begin by remembering our past mistakes in evaluating Jesse Gelsinger who was 17 when a geneticist told him that his OTC deficiency disorder, ornithine transcarbamylase deficiency, which is an inherited disorder that allows ammonia to accumulate in the blood, could be cured with gene therapy. One day during his treatment he started vomiting uncontrollably and he ended up in the hospital on life support. The vector used to help his immunodeficiency order had deleterious side effects and caused him to have jaundice, multiple organ failures, to be brain dead, and

Gene therapy is a technique that uses new genes to treat or prevent disease by replacing or adding new effective genes. This technique is aimed to allow doctors to treat genetic disorders without the prescribing drugs or scheduling surgery to correct the disorder. Gene therapy isn’t a fully effective method as of today. It is still undergoing tests. Several of the most common tests include introducing a new gene into the body to fight against a disease or disorder, replacing a mutated gene with a healthy gene, and removing a mutated gene in the body that is not functioning as it should be. Gene therapy is seen to be a fair treatment to diseases or disorders that have no other form of cure. It is sometimes used for inherited disorders, certain viral infections, and certain cancers. But Gene Therapy is not a certain option for treatment as of today. It is still under study as success rates are low. Genetic disorders affect one out ten people. Symptoms vary with each disease or disorder as some can appear early in a person’s life, while others appear at an older age. Genetic disorders cannot be prevented as they are passed on through a family’s genes and is within the DNA unlike a regular disease is caused by bacteria or a virus. There are two possible ways a genetic disorder can be passed on to the offspring, or child. One way is caused by dominant genes in which only one gene is altered resulting in the disorder or disease. The second way is caused by recessive genes in

Gene is a relatively new but rapidly developing way to treat or cure people of certain diseases. Instead of using a drug or surgery to cure or treat someone they are using gene therapy as a way to insert a gene into the patients cells. If this technique works then it can be an amazing option for someone with a disease with otherwise no cure. Along with this new technique come many risks and dangers that question the ethics involved in doing this. Even though it is only still being tested it can be seen as a promising treatment option for many people.

disease (NLM, 2014). The procedure uses a corrected gene and inserts it into a person instead of