The Controversial Summary Of 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).

Gene therapy is an experimental technique that is used to prevent disease. For future reference, this technique may treat disorders. It may add or remove genes to get rid of a genetic disorder. Gene therapy is a heavily debated subject. There are very respectful points on both sides of this debated topic. The pro side believes that gene therapy is the best thing that is happening to our society. However, opposers of gene therapy believe that people should refrain from proceeding with this biological advancement and it is ethnically incorrect.

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

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

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

In the medical journal article “Harlequin Ichthyosis”, the authors Claire A. Scott, Shefali Rajpopat, and Wei-Li Di explain how “ABCA12 mutations underlie defective lipid transport, reduced protease regulation and skin barrier dysfunction." The first known case of HI was discovered in 1750, and was documented in the diary of Reverend Hart. The effects are apparent at birth and are commonly clinical. The entire body is born covered in a compact layer of white plates of scales separated by deep red cracks. The thickened skin, usually restricts movement and causes limb contractures, inevitably causing digital necrosis and autoamputation.

This hotly debated topic of whether it is dangerous “playing God” has caused many religious, political, scientific, and public figures to express their concerns in heated debates (Hogarth para 9). Ever since the beginning of gene therapy, there has been constant dispute and friction over the direction of its future; of whether gene therapy will evolve into a therapeutic science or lead to horrible consequences as the eugenics movement did in the 20th century. The ethics of gene therapy have been based on, not only the beliefs, but also the fallbacks and advantages gained in experimental trials, which has inevitably lead to the progression of the future of gene therapy.

We currently group types of gene therapy by the nature of the cells which are affected and the goal of the therapy. Nelson Wivel, director of the Office of Recombinant DNA Activities at the National Institutes of Health (NIH), and Georgetown University professor of philosophy and ethics Leroy Walters (1993) group types of gene therapy into three categories: type 1 is gene therapy which affects only non-reproductive or somatic cells, type 2 is gene therapy which involves gene transfer to reproductive or germ-line cells, types 3 and 4 are the use of somatic and germ-line modifications respectively to "affect selected physical and mental characteristics with the aim of influencing physical appearance or abilities" - enhancement therapy (p. 533). In the U.S., the Recombinant DNA Advisory Committee (RAC) at NIH oversees research and clinical trials of gene therapy. At present the RAC permits many somatic cell therapy trials but does not permit germ-line

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.

The objective of gene replacement therapy is to alter the defective gene to achieve a permanent cure for the diagnosed patient by identifying the faulty gene and providing a corrected copy of the gene without affecting the genomic sequence. Clinical trials initiated gene replacement experimentation, primarily leading to significant progress but not yet accomplished.“The most recent clinical trial for the treatment of hemophilia with gene transfer showed transient achievement of efficacy in the highest dose cohort tested, but also exposed a previously hidden barrier to the future success of these treatments.” (Gene Therapy for Haemophilia) Recently, the focus of gene therapy strategies is mostly on gene addition rather than gene replacement.

Deoxyribonucleic acid (DNA) was discovered in 1944 by Avery and colleagues. Avery identified DNA as the primary genetic material. Watson and Crick later discovered the double helix structure of DNA. Leder and co-workers deciphered the triple nucleotide code that designated the amino acids from which proteins were built. The science of molecular biology was born (Sokol, Gewirtz, 1996). In 1990 a four year old girl who was suffering from severe combined immunodeficiency (SCID) was the first to undergo gene therapy. White blood cells were removed from the girl and the cells were inserted with normal copies of the defective gene and returned into the girls circulation. Her condition improved with four treatments and

The word science comes from the Latin word "to know" (Science Made Simple, 2014). In

Gene therapy techniques consist of several key elements. An important element, actually one of the most important elements is the identification and cloning of the gene or genes related to the disease, which is going to be maintained or treated by gene therapy. However, gene therapy should not be confused with the concept of cloning. Cloning is creating another individual with essentially the same genetic makeup. The idea of cloning an entire human is very different from gene therapy. Next the expression and efficiency of gene transfer during gene therapy must be emphasized. Scientists involved with gene therapy research are currently trying to discover the best rates of gene transfer and tissue-specific gene expression. Gene therapy research is still in the formative stages. Although a number of early human clinical trials have been initiated to test important concepts that have emerged.

There is only one viable technique in gene therapy to humans – adding a gene to replace improperly functioning gene through a vector. Its basic mechanics is administering the DNA to the patient. Introduce the DNA to the diseased cell by a genetically engineered virus and express a protein. This can be done by injecting it to the bloodstream. The therapeutic genes are hoped to genetically alter the cells for normal functioning.

Gene therapy is described as the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders. 1 During the 1960’s and early 70’s the actual concept of what is now known to be gene therapy arose. Many new practices including the development of genetically marked cell lines and the delineation of cells transformation by the papaovaviruses polyoma were in the works. Cloned genes became a product of this new DNA technique and were used to prove that foreign genes could actually correct genetic disorders.2 This new technology in the medical field has a wide range of uses that is constantly growing larger as scientists continue to study and experiment with it. As of right now, the uses of gene therapy in the medical field consist of replacing missing or defective genes, delivering genes that speed the destruction of cancer cells, supplying genes that cause cancer cells to revert to their normality, delivering bacterial or viral genes as a form of vaccination, providing genes that promote or impede the growth of new tissue and delivering genes that stimulate the healing of damaged tissue.3 With this information, a wide variety of genes are now being used for testing within gene therapy. In the mid 1980’s the first use of gene therapy was practiced and then seen in a four-year old girl when she became the first gene therapy patient on September 14, 1990 at the NH Clinical Center. This four-year