Essay about Priotities of Gene Therapy

Akudo Ejelonu writes, “The medical industry is building upon the knowledge, resources, and technologies emanating from the Human Genome Project to further understanding of genetic contributions to human health....Genetics is playing an increasingly important role in the diagnosis, monitoring, and treatment of diseases” (What is the Human Genome Project-Why Is It Important To Society par.10). According to this evidence, the Human Genome Project has opened doors for scientists to better understand the use of gene therapy for humans. Genetics is now playing an important role in curing, monitoring, and diagnosing diseases that can make someone very sick. With gene therapy it can save lives and help cure the world of its deadly diseases. “Genetics Home Reference” states, “ Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS” (Gene Therapy Experimental Techniques, Safety, Ethics, and Availability par.15). The “Genetics Home Reference” is insisting, gene therapy is on its way to becoming a treatment for anything and everything. It is on its way to becoming a full time treatment for diseases that will later be cured because of frequent gene therapy sessions with sick patients. All of the hundreds of research that has been done on this subject is just a grasps an understanding of how it all works and how it changes the world in a positive way. Larissa Hirsch states on page three, “Scientists hope that the human genome mapping will help lead to cures for many diseases and that successful clinical trials will create new opportunities”(Gene Therapy and Children par.4). Larissa Hirsch is stating that the Human Genome Project plays a big role in gene therapy. The Human Genome Project is opening doors for scientists that have successfully performed medical trials when it comes to treating

Gene Therapy is an experimental form of treatment that aims to get rid of genetic conditions at their source. It also may be used to replace a faulty gene with a working version or introduce a new gene to cure a condition or modify its effects. ( Victoria State Government, 2011) It is designed to introduce genetic material into cells to compensate for abnormal genes or to create a beneficial protein within the gene. If a mutated or incomplete gene creates a protein needed that is faulty or not present, gene therapy has the ability to introduce a copy of that specific gene to restore the function of the protein within that gene.

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

(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.

Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy.

As years go by and technology advances, many medical techniques that failed in the past are starting to seem more possible. Researchers are discovering that a technique called gene therapy is able to help prevent and reverse the effect of many conditions. Originally in the early 1900's, gene therapy failed in

According to Blau and Springer (1995), gene therapy is a newly rising approach at the end of the 20th century and aims to open new door for the treatment of various human diseases based on transfer of genetic materials to cells. The recent gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy is not inherited and thus it cannot be passed down on to a person’s children in the future. Meanwhile, gene therapy could also be targeted to egg and sperm cells (knowns as germline gene therapy) which would allow the inserted gene to be passed on to the progeny. Germline gene therapy allows the correction of disease-causing

Within this field, somatic gene therapy—the replacement of a faulty gene with a functioning one—is particularly fascinating. One of its interesting aspects is the ability to be customized to the individual. For example, not all patients suffering from a given cancer will have the same mutations. After sequencing a patient’s genome to detect the carcinogenic mutations, doctors can use gene therapy to target and treat the illness at its source. However, at this stage of the technology, gene therapy provides only short-term remission. Studies on blindness showed that the initial success of gene therapy faded over time—an unsurprising outcome since the cells of most tissues will die and be replaced by new, untreated ones. With this area of research still in its infancy, much remains to be done to improve the long-term effectiveness of gene therapy and make it a viable treatment. A potential solution would be to use gene therapy on stem cells as well as on specialized cells. The daughters of the treated stem cells can then differentiate, still carrying the therapeutic gene, and replace the original treated cells as they senesce. For so long, genetic disorders have been seen as something permanent, something scientists could not change. Changing that reality would be immensely

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

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

Throughout history humans have been able to manipulate various objects and situations in order to further advance society towards an ideal vision. One particular procedure that has gained a significant role towards the importance of the medical society applies to gene therapy and how it has certainly produced an extensive amount of beneficial accomplishments. Gene therapy generally consists of regular genes being switched into a cell in order to replace its defective genes. Introducing a variety of distinct genes into cells of a body can highly raise the risks of them actually remaining functional due to rejection from the body. Due to this process, gene therapy contains a very critical position in the challenges of modern medicine ().

Gene therapy is a relatively new concept, it is an experimental technique that uses genes to theoretically treat and prevent various types of Illnesses/Genetic Disorders such as Haemophilia, Cancer, Chronic Granulomatous Disorder and Cystic Fibrosis. The process of gene therapy involves the use of a Viral Vector (Most Common Form) other forms of delivery which are non-viral include the injection of naked DNA, sonoporation and magnetofection.

Gene therapy is intriguing to discuss and research because it’s untapped potential that it has and the ability it has to cure and eradicate countless genetic disorders such as Alzheimer’s disease, Parkinson’s disease and even cancer! It also has endless possibilities that it could contribute to the health industry. How far gene therapy has come and how far science has come, for this to even be possible is

But many view these risks as subordinate to the potential of gene therapy to help reduce and eliminate the social burden of genetic disorders. The ability of gene therapy to affect such disorders,however, is restricted to those caused by recessive X-linked or autosomal mutations. The realm of efficacy for treatment includes individuals afflicted with common disorders such as haemophilia, color blindness, Duchenne muscular dystrophy, phenylketonuria (PKU), cystic fibrosis, sickle-cell anemia, and Tay-Sachs disease(226-7). The treatment of such disorders must be monitored so that the replacement gene, once inserted, will be expressed at the appropriate time and place, and in the correct quantity(229). Improvements on regulatory techniques are then necessary to enhance the effectiveness of gene therapy. Furthermore, the effectiveness of such techniques is exceeded by the vast majority of the population afflicted with genetic disorders caused by

For example, gene therapy can be used to cure genetic diseases like leukemia. For instance, a young man from Douglas, Arizona, a town boarding Mexico, underwent chemotherapy, but if he had gone through gene therapy instead of the harmful chemotherapy radiation that could have been performed many toils would have been prevented for the young man and his family. Gene therapy is an experimental way of using inheritable factors to treat or prevent diseases it is also an experimental technique that is used to substitute a gene that works right for a non-working gene. In fact, in human genetics, gene therapy is the use of genetic material as medicine involving the transfer of working or therapeutic copy of a gene into specific cells of an individual to repair a copy of a faulty gene. In the future, this technique may be used to allow doctors to treat any disorder instead of using drugs or surgery by inserting a gene into a patient’s cells. Several researchers are testing numerous approaches to gene therapy, including “taking out” or deactivating a mutated gene that has improper functioning, and introducing a new genetic factor into the body that will help fight a disease. In fact, several areas of criteria must be met for the procedure to work.