Gene Therapy : An Experimental Technique

Gene therapy: Is a treatment method that involves the manipulation of an individual's genetic makeup. A form of therapy that attempts to fix the defective gene which is causing the disease.

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).

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.

Some people may wonder what gene therapy is. Gene therapy is the replacing of defective or missing genes with normal genes in order to cure the disorder. In the future, this technique may be used to prevent or treat a disorder by inserting

Gene therapy is a method involving DNA to treat or prevent disease. It is the insertion of a corrected gene into an organism’s cell to mask the presence of the dysfunctional gene, instead of surgery or using drugs. Put simple, gene therapy is the process of using DNA as a drug. Generally, a vector (‘delivery vehicle’) is used to go to the cells where it is needed and deliver a gene, if this is successful, the cell will then be able to produce a functional protein to treat or prevent the disease. Since the gene causing CF was discovered, it is now possible to attempt gene therapy to

Gene therapy (otherwise known as somatic gene therapy or therapeutic gene therapy) is a process that is used to correct defective genes responsible for development of various diseases. (www.accessexcellence.org/RC/AB/BA/Gene_Therapy_Overview.php) There are two main types of gene therapy; somatic (body) cell gene therapy and germ line gene therapy. As it suggests, somatic gene therapy is correcting the faulty genes of normal body cells. Germ line therapy is altering genes in gametes. Everyone on earth carries a few defective genes. Most of us are unaware of this fact however, unless we are afflicted with a genetic disease. Approximately one in ten people have, or will develop, a disease that is

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

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 is also a good technique for diseases not researched yet. All of us carry defected genes and may not know it. Gene therapy is a 'medicine" for the future since it can control or eliminate hereditary diseases. This means that Gene Therapy is a good source for our future and will help us prevent diseases that have not been looked at more closely.

Good afternoon Mr David and class. Today the topic of gene therapy will be discussed, so to begin, what is gene therapy? Basically, gene therapy is the modification of genes to alter a certain characteristic that ultimately can cure a genetic disease. There are two types of gene therapy: somatic gene therapy and germline gene therapy. Somatic gene therapy is when blood cells are extracted from a person and a normal gene is then inserted into the defective cell. This does not prevent the disease from occurring in future generations because it doesn’t affect the sperm and egg cells, only the body cells. Although, this is a treatment that has to be repeated several times throughout a person’s life as the effects can wear off. Germline gene therapy however occurs in the reproductive cells where there is genetic modification to these cells occurs. Because these are reproductive cells that are being modified, these changes WILL be passed down to the next generation. Contrasting to somatic gene therapy, germline therapy only has to be done once in a lifetime, as it is permanent.

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:

This technology can target specific diseases on genes such as Alzheimer’s, Parkinson’s, polio, cystic fibrosis and the list can go on forever (Hackett, 2013). A popular method used to implement new genes is the adenovirus. A gene cannot simply be integrated into cells, it must have a vector, or a carrier which in this case is the adenovirus which helps integrate the gene into the DNA of the targeted cells (NLM, 2015). Another option is available where the subject’s cells are removed and injected with the gene and reinserted into the body (NLM, 2015). Both methods have shown success. Recently, in Oxford retinal disease has been overcome by using gene therapy (McClements, 2013). Several cases in this study allowed men and women to overcome a range of retinal diseases and avoid blindness and this is all due to the amazing effects of gene therapy (McClements, 2013). They used the adenovirus as a vector to deliver the new genes into the retinal cells in order to make the change in the eye. It was a hard fought success. There are several cases just like this study that have allowed people to renew their lives through human gene

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

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

Genetic Therapy includes the identifying of a broken or bad gene in an organism and then the insertion of a good and working gene into the organism. The idea is that the subject’s body will accept the DNA and as it duplicated it will outnumber the bad gene, therefore