There are also difficulties in getting the targeted cells to open up their molecular locks to allow the foreign genes inside. Gene therapy, like other medical advances before it, will have numerous failures before reaching its full potential. It will be important for the public, press, and medical industry to be patient in waiting for the dream of gene therapy to become a reality. Technological Aspects of Gene Therapy The underlying principle of gene therapy is the transfer of genetic material to specific cells of a patient in an effort to initiate a biological response to fight or eliminate a disease. There are two possible types of target cells, somatic cells that are non-reproducing, or reproducing germ-line cells.
Gene therapy is the insertion of normal or genetically altered genes into cells usually to replace defective or diseased genes in the body. By using gene therapy, doctors can go to the source of the disorder or problem instead of prescribing a patient a countless number of drugs (Hogarth 1). Gene therapy was designed to introduce new and corrected genes to compensate for any abnormal genes. If a mutated gene causes a protein to be missing or to fail, gene therapy is a way to inject a new and normal copy of the correct gene to revitalize the function of the lost protein (Genetics Home Reference 1). With the help of medical advances in technology, gene therapy has gone from the idea stage, to technology development and laboratory research, to clinical trials for various disorders (ASGCT 2).
Gene therapy is revolutionising the world of medicine and it could be the biggest and most helpful treatment created. Gene Therapy is designed to insert genetic material into abnormal genes. When a gene is inserted direct to cell it will not function so a carrier 'The Vector'- a genetically engineered DNA molecule is used to deliver and welcome the new type of gene into the body to fight the disease. A mutated gene can cause the protein that is needed to be missing or faulty, Gene Therapy can introduce a healthier and brand new copy of the gene to reset the function of protein. A virus is used as a vector, delivering the gene by being inserted directly into the specific tissue in the body, where it is taken up by individual cells.
There are many moral and ethical decisions that need to be considered before gene therapy can be widely accepted. Do we have the right to change a person’s genetics, especially before they are born? Do we know enough to confidently insert or delete genes without detrimental consequences down the road? If we have the ability to help people who have disabilities or diseases, is it ethical to withhold and not treat the patient? I believe human gene therapy is a good and useful tool for medicine and needs to be developed because it posses the ability to help and cure people from ailments that degrade their quality of life.
Gene therapy will change the field of medicine from what it is today. As scientist discovers more genes and their functions, the potential of this treatment is limitless. Though gene therapy is an auspicious treatment choice for numerous diseases (including inherited disorders, some types of cancer, and certain viral infections), the procedure remains precarious and is still under study to make sure that it will be safe and effective. Thus government regulators and scientist must take a lead role in adopting a practical approach to address these issues and determining the correct procedures for dealing with them.
As stated earlier in the paper there is different approaches to how gene therapy could be used. Examples given earlier were; swapping a mutated gene that causes a disease with a healthy version of the gene, deactivating a mutated gene that operates incorrectly, and having a new gene introduced to the body to assist in fighting a disease. Therefore, for those who believe and agree with all the pros and benefits of gene therapy would have a great deal to look forward to. (1) (3) Future effect Researchers could have a cure to diseases that were incurable. Gene therapy has the possibility of being able to alter genes in an unborn child.
DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line therapy is very similar to somatic gene therapy with the exception being the therapy of somatic cells instead of sperm or egg cells. Some of the pros of gene therapy are that it is only way of curing genetic disorders so it is our only hope for finding cures for such disorders. Once gene therapy treatment becomes safer and effective it could wipe out genetic disorders as cystic fibrosis, Parkinson’s disease and cancer completely. This is because gene therapy targets the reproductive cells of carriers of such genetic disorders so it is possible that any children the carrier goes on, gene therapy would free them of the defective gene (Ali et al., 1994).
There are two types of gene therapy treatments. They differ in the consequences they have for the patients. The first is somatic cell gene therapy. In this process faulty genes are replaced by the correct sequence of genes. The body can then make the correct protein and ultimately eliminate the faulty gene causing the disease.
Gene therapy has become an exciting and controversial issue on the scientific and medical horizon. Science offers new technologies that, in the future, will be able to treat and cure common genetically passed diseases. However, as it is an extremely broad subject, some time must be dedicated to its interpretation and explanation. First, a general definition of gene therapy is required. Genethics, the Clash between the New Genetics and Human Values, by David T. Suzuki and Peter Knudtson, defines gene therapy as "the medical replacement or repair of defective or faulty genes in living human cells."
Gene therapy is a highly controversial topic that entails numerous ethical issues that need to be thoroughly analyzed before it is widely available to the public. While gene therapy may pose practical medical benefits for people, ethical considerations must be addressed in order for society to utilize the potentials of gene therapy appropriately. What is gene therapy? Gene therapy attempts to cure or treat genetic diseases by correcting the genetic errors responsible for it. Genetic diseases can be either inherited diseases such as cystic fibrosis as well as acquired diseases such as cancer ("What is Gene Therapy?").