Genetic Disorders: 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).

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.

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

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

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

Gene therapy is a technique that allows for the modification of genes to treat or prevent disease and may become a viable way for doctors to treat a disorder without the need of drugs or surgery. This technology is still experimental, being only about three decades old with the first successful human trial occurring in 1990 when Dr. William French Anderson treated Ashi DeSilva for Adenosine deaminase deficiency (ADA deficiency), which is a genetic, metabolic disorder which causes immunodeficiency. Researchers are testing several approaches to gene therapy, which include replacing mutated, disease-causing genes with a healthy copy of the gene, inactivation a malfunctioning gene, and introducing new genes into the body that will help fight diseases. Over the last two decades, about 2,000 trials have been conducted or approved and future prospects for this technique are encouraging, include the engineering of human genetics in ways that may prove controversial and difficult to predict. This paper will explain how gene therapy works, what it has accomplished so far, its future and current hurdles, and what this all means for our species.

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

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.

Hemophilia A was a fatal disorder. However, its treatment progressed rapidly around the middle of the last century when patients were infused with plasma or processed plasma products to replace Factor VIII. Around the mid 1980’s, the development of alternate Factor VIII sources for replacement therapy was discovered including monoclonal antibody purified Factor VIII and recombinant Factor VIII, both of which are used in replacement therapies.

Moreover, Positive aspects of gene therapy are apparent as it can give many peoples a new life. It can reduce the stress on future parents as they do not have to think or worry about having a child with disabilities they can have a normal child because gene therapy can wipe out genetic disease before they can begin and eliminate suffering for future generations; it is also good technique for diseases that are not searched yet because each of us caries about half a dozen defective genes, however we remain ignorant to this fact unless we are among the millions of people who have a genetic disorder; about one in ten people has or will develop some time later in life an inherited genetic abnormality. It and can also silence a gene in the case

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

Researchers have been trying to find new ways to cure disease for centuries. Gene Therapy

The world of genetics has mainstreamed into medical practice, transforming the way patients are treated. Recently, gene therapy has been propelled into the spotlight with the most recent successful clinical trial in which scientists reprogrammed ordinary cardiac cells in pigs to mirror the function of the sinoatrial node, thereby inducing a steady heartbeat. In the last few decades since its inception, gene therapy has rapidly advanced; there have been remarkable strides in its methods and an increase in the range of targetable diseases.