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.
The definition of gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases (Jaroff, 1996). Gene therapy has two possibilities of disease treatment, somatic gene therapy and germline gene therapy. Somatic gene therapy involves the manipulation of gene expression in cells that will be corrective to the patient but not inherited to the next generation. Germline gene therapy involves the genetic modification of germ cells, which pass the change on to the next generation (Wilson, 1998). Somatic gene therapy is currently being researched more aggressively due to ethical and technical complications with germline gene therapy.
Critics may argue that there are moral and ethical problems associated with this novel technique, but for the most part scientists realize the importance this advancement will have. Gene therapy may be the key to curing dozens of diseases, and has endless possibilities, but more research is needed before its safe or accepted as common practice.
“ Gene therapy is the insertion of a functioning gene into the cells of a patient to correct an inborn error of metabolism (i.e. genetic abnormality or birth defect) to provide new function in a cell (e.g. insertion of an immunostimulatory gene into cancer cells to vaccinate a patient against their own cancer) (Culver, xvi). There are currently two types of gene therapy. Somatic gene therapy, which is currently the only accepted method of gene therapy for use in humans, is the changing of all the cells of the body except for the reproductive cells.
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.
¡° To obtain correct gene action, it may be necessary to put it into the correct site on the host cell chromosome, or even to delete the defective gene¡±, and the DNA can then be replicated each time the host cell divided. But if the new cell is injected directly into the patient¡¯s body, it will be subject to the body¡¯s immune system that will recognize it as foreign and target it to be destroyed along with the healthy DNA that it is carrying. So the cells extracted from the patient are to be treated and adding the new gene in a test tube in the laboratory to make sure that the DNA is inserted in an appropriate place in the genome, and the cells can then be returned to the patient¡¯s body. Now it is possible to offer the parents an antenatal diagnosis to look over if the fetus is affected by some single gene defects. If it does, the parents can choose embryo therapy to cure it rather then abortion.
Human Gene Therapy: Gene Therapy can be defined as an experimental technique that uses genes to prevent or perhaps treat diseases. It works out by allowing doctors to treat the disorder by inserting a gene into a patient’s cell and not using surgery or drugs. There has been several approaches to gene therapy such as; launching a new gene into the cell to help fight a disease, inactivating a mutated gene that is doing harm to the body, and replacing a defective gene with a “corrective” copy of that particular gene. Gene therapy could have the possibility of treating numerous diseases but it is currently being tested for diseases that still do not have a cure. It was design to introduce new genetic material into cells to help “correct” mutated genes or help the cell produce a necessary protein.
Gene therapy is a relatively new form of treatment, one that is still being experimented and researched. It is the use of genes to treat diseases by inserting them into cells either to replace a mutated gene, to inactivate a mutated gene, or to fight against the disease. (Genetics Home Reference, 2014) As with any medical treatment, the advantages should be weighed against the disadvantages before going through with it. There are two types of gene therapy: somatic gene therapy and germ line therapy. Somatic gene therapy is when the body cells, excluding the reproductive cells, are altered.
In cells with dominant negative genes, resulting protein must be prevented from translation or transcription using oligonucleotides or antisense technology. Though many obstacles delay the development of gene therapy, this new field will surely improve the future of medicine. Mechanism of Transfer in Gene Therapy Gene therapy is a method of replacing defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function.
In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes.