Gene Therapy Essay

Gene therapy has saved many lives and given a second chance to many people. Gene therapy was used to treat a girl 6 years old named Emma who was suffering from leukemia. The doctors had tried many treatments to cure Emma from cancer and nothing was working even though, kids at her age have an 85% chance to survive. She was on the verge of death when gene therapy was used to alter Emma’s immune cells to genetically kill cancer cells. Emma has been cancer free for two years and is living the life of a normal child.

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.

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

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.

Humans have been able to use the principles of DNA replication, gene transfer and gene expression (as observed in nature) as tools to manipulate specific genes towards preferred outcomes. An advantage of being able to manipulate genetic therapy is the ability to eliminate/cure genetic disease/disorders with otherwise no other cure. Humans are able to remove the genes causing the genetic disorder from the gene pool of populations, allowing organisms to live longer and healthier lives. Two ways of manipulating genetic transfer which will be explored further in this report are selective breeding and gene therapy.

Roughly fifty years ago, scientists thought that the use of genetic modification by exogenous DNA would be effective. This idea could offer curative and long lasting benefits, in a single treatment.

Without a vector, mutated cells cannot be targeted, which means the treatment would be ineffective. Since the 1990’s, scientists have experimented with the use of viral vectors, such retroviruses, adeno viruses and adeno asscociated viruses. Viruses contain a circular piece of DNA known as the plasmid. During gene therapy, the segment of DNA responsible for viral spread is removed. The correct copy of the mutated gene is then inserted into the virus. The virus is then inhaled,swallowed or injected depending on the location of the target cells. (in the case of CF, the vector is inhaled). Retroviruses are viruses that contains RNA instead of DNA. Retroviruses can only infect dividing cells and the viral RNA needs to be reverse transcribed to DNA, so that gene can be replicated for mitosis (this is done using enzymes). Unfortunately, retroviruses have been found to implant genes into random parts of the genome. This means that the introduced gene can disrupt an existing, functional gene. Between 1999 and 2006, reasearchers performed gene therapy on 5 children with a genetic immune defficiency, using retroviruses as vectors. By 2006, all 5 of these children had developed leukemia as a result of the vector implanting into a random part of the genome. Adenoviruses have also been used as vectors, however, they do not integrate into the genome permaneatly and are likely to trigger an immune response. In 1999,

Gene therapy is a relatively new technology that has shown some promising results. Such as the drug called Strimvelis that is used to help children with severe combined immunodeficiency, a disease that leaves infants with little to no protection against viruses, bacteria, fungi, and other potentially harmful things. It appears to be a cure carried out through a genetic pair. Strimvelis was tested on 18 children, starting 15 years ago, all of whom are still alive today. Gene therapy is used to inject genes into a person’s cells and tissues in order to treat a disease, as well as hereditary diseases where an abnormal allele is replaced with a fully functional allele. The carrier, called a vector, is used to transport the normal gene to the target cells. The most common type of vector are viruses that have been genetically altered to carry human DNA. Scientists have been trying to control the way the viruses have evolved encapsulating and delivering their genes to normal cells in a pathogenic way by manipulating the viral genome to remove disease-causing genes and inserting healthy ones. Theoretically, it is possible to transform either the cells of the body (somatic) or the cells of the sperm, ova, and their precursors (germline). All of the gene therapy experiments so far on humans have been directed to somatic cells, because germline experiments have been remaining controversial. Somatic gene therapy is split into two broad categories: ex vivo (cells are modified outside of the body then returned back again) or in vivo (genes are changed in cells while still located inside of the

Despite the promise of such approaches, a number of difficulties remain to be overcome, the most important of which is the need for more efficient systems of gene delivery. No gene transfer system is 100% efficient, unless germ-line therapy is contemplated. During the past two decades, there have been major advances in our understanding of how cancer develops, proving that cancer has a genetic basis (2). A series of genetic abnormalities that accumulate in one cell may result in a pattern of abnormal clonal proliferation. Our growing understanding of the genetic basis of cancer offers new opportunities for the molecular prevention and treatment of cancer. There has been a

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).

The procedure consisted of taking a piece of the persons liver and injecting correct copies of the genes into the flawed liver cells, and placing the piece of liver back into the person. Two years later, the results showed that the corrected cells were thriving. As a result to the cells, the person had reduced their cholesterol by twenty percent.

If a treatment is implicated before the child is born – germline gene therapy – he or she could lose all trace of that defective gene, and therefore wouldn’t pass the disease on to future generations. In somatic gene therapy, treatment is conducted when the patient is an

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

    Through recombinant DNA techniques, scientists have already experimented with bacteria and copied their cellular system that allows them to defend themselves from viruses in order to create a new restriction enzyme called Cas9, which then gets bound to an RNA guide strand. This new enzyme introduces new genetic material into the human genome as DNA replicates. This method will be useful for families members who carry harmful recessive alleles. Their babies could inherit the genetic disorder, but with recombinant DNA techniques, the harmful gene or genes could easily be replaced with a functional gene. Lauren Friedman, Senior editor for

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