The Discovery Of Gene Therapy

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

genes or adding new genes to cure or prevent disease (National Academy Press). This news

In one gene therapy study, scientists are investigating the use of a type of skin cell, keratinocytes to see if they are a successful option to prevent the gene that expresses faulty keratin, from successfully expressing. Another study shows that researchers have found a way to successfully deliver cells that have been modified back into the living host (National Institute of Health, 2013). This test has only been successfully done on mice so the results in human organisms are not available. Scientists are now testing to see if this gene therapy will also work on humans and how long the process lasts. Scientists are further exploring techniques that will help find the precise moment when the gene mutates during development so that a possible development of a cure for EB will be found in the future (National Institute of Health,

Human gene therapy is when genetic material is introduced into human cells to treat a genetic disease. Instead of traditionally treating the symptoms, gene therapy aims to treat the disease’s root cause by directly altering genes and correcting defects. Therefore, gene therapy is only viable for certain diseases. Disorders from mutations in single genes are the most treatable, whereas disorders that involve many genes and environmental factors are trickier.

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

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).

Gene is a relatively new but rapidly developing way to treat or cure people of certain diseases. Instead of using a drug or surgery to cure or treat someone they are using gene therapy as a way to insert a gene into the patients cells. If this technique works then it can be an amazing option for someone with a disease with otherwise no cure. Along with this new technique come many risks and dangers that question the ethics involved in doing this. Even though it is only still being tested it can be seen as a promising treatment option for many people.

Gene therapy is a field of study that focuses on the correction of mutations in DNA sequences. The ultimate goal of Gene Therapy is to prevent the inheritance of mutations that cause genetic disorders. In the medical community this is called Germ-line Gene Therapy. While this is the least ventured area of gene therapy, its has been proven to be the most promising in reference to advancing medicine to new heights when the ethical situation changes. The second  goal of Gene Therapy is to cure the patients with the genetic

Gene therapy is processes of transferring DNA in to a drug as a treatment for certain diseases. Gene therapy was for discovered in the mid 1970’s and it is simply developing a therapeutic form of DNA and putting it into a patient’s cells. In other words this experimental technique in other words uses genes as a treatment or as a prevention to certain diseases. The why this works is that genes are passed down through inheritance. Meaning they go from a parent to its child and then to the child’s child etc. However a certain gene does not produce the right protein or does not produce a protein in a correct manner then a genetic disorder is formed. To dealt with many of these diseases scientist and doctors have been treating the symptoms of the disease but they do cure the actual disease. In other words what they have been mopping and drying the floor from a leak instead of fixing the actual pipe that causes the leak. However gene therapy would be the perfect way for both scientist and doctors to find a way to cure a disease or fix the pipe rather then the symptoms.

Having a cure for genetic diseases would be a significant progress in the field of medicine. [3]

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.

In initial stages, gene therapy was used to treat patients with only life threatening diseases and illnesses; however is it now used to treat non-life-threating diseases too as a means to improve the standards of life of patients. Depending on the type of cell being treated, there are commonly two different types of gene therapy used.

Gene therapy is a technique that uses genes as medicine to treat or prevent disease. The technique may be used to replace a defective gene for a healthy gene or use a new gene to favorably change the condition of the faulty gene [1]. There are several problems that challenge gene therapy, the certain condition or disease in question must be well understood, the defective gene must be identified and a working copy of the gene must be available, and the most problematic is effectively delivering working copies of the gene to the cells. The DNA delivery vehicles used in gene therapy are called vectors. There is no omnipotent vector that can treat every disorder. Every gene therapy vector must be customized to address the unique features of the disorder [3].

Gene therapy is a technique that is used to cure diseases, and to stop malfunctioning genes from producing the disease in humans. Gene therapy involves introducing normal functioning genes into a sequence containing malfunctioning genes, which either stop or hinder the production of important proteins or cells needed in the body. Doctors are able to cure diseases, and may soon be able to prevent diseases, by taking copies of the DNA sequence with the malfunctioning gene, and inserting a better working gene into the DNA sequence, which will then provide the cells with the correct amount of proteins that it needs to carry out bodily processes. The most common techniques of gene therapy used in order to cure severe combined immunodeficiency diseases are, Restriction enzymes, PCR, DNA sequencing and Gel electrophoresis, Ligation and viral vectors. These four techniques allow scientists to insert functioning normal genes into the area causing the disease, and produce the correct amount of proteins needed to cure humans.

During the 1970’s period, the accepted scientific view of gene therapy was optimistic for future treatments of human diseases, specifically hereditary ones such as sickle-cell anemia or cystic fibrosis.