Human Gene Therapy

One must also consider the ethical questions that arise. Gene therapy offers undeniable benefits, but the risks it poses need to be addressed before this technology can become common practice. The goal of gene therapy is to correct the unwanted trait or disease by introducing a modified copy into the cell. Notice that the purpose is not to replace defective genes in the host cell, rather it is to provide a new copy, so the correct protein is expressed, or at least the defective gene is neutralized (Blachford 462). Humans are made of trillions of cells, each with a specific function.

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. Alternatively, a sample of the patients cell can be removed and exposed to the vector in a laboratory. If treatment it successful, new gene delivered by the vector will make a functioning protein. Researchers and scientists have many technical trials before treating a patient. However through the whole process the body must stay in control.

Gene therapy is the delivery of a correct version of a mutated gene to a cell, where its expression will produce the normal protein and restore normal cellular function. The mutated gene must be delivered to the nucleus of the cell. (2) There are essentially two forms of gene therapy, 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 by the next generation. (4) Germline gene therapy involves the replacement of defective genes in the germline cells, which contribute to the genetic heritage of the offspring.

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.

¡° 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.

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.

There are a few methods of altering cells in gene therapy. The first method is in vivo. The in vivo method is where the cells are altered inside of the body. For the in vivo method to work accurately, there needs to be a certain way for the genes to reach the target cells (a ve... ... middle of paper ... ...ays inside of the cell until it is evicted by the body’s immune system. Cancer can be cured with gene therapy by many methods.

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.

The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them in a laboratory. Thus the corrected copy of the gene is inserted into the cells before being put back into the body. Gene therapy can also be done in vivo which can be done directly to the patient’s body. The word gene therapy really defines the management of genetic information that is encased in the cells, however, in most recent procedures the available technology is closely related in adding new genetic information, and many researchers favor the term gene transfer rather than gene therapy to mirror the reason that the purpose of gene work cannot always be therapeutic.

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.