Gene Therapy is a modern treatment which uses genes to treat or prevent genetic diseases. It is a process of which DNA is used as a drug to treat a disease by delivering therapeutic DNA into a patients cell. The mutated gene is replaced by a healthier, functional/therapeutic gene which corrects and converts the cells lacking in protein or faultiness. Our future is based behind our genes in the body and scientists are trying to move away from drugs as a solution. Gene therapy is revolutionising the world of medicine and it could be the biggest and most helpful treatment created.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases. For example, suppose a brain tumor is forming by rapidly dividing cancer cells. The reason this tumor is forming is due to some defective or mutated gene. The therapy chosen for this case would be to use a herpes virus that has had its virulence removed, rendering it harmless. The virus is still abl e to insert its genetic material into the target 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.
Gene splicing involves removing a fragment of DNA containing the specific DNA sequenc... ... middle of paper ... ...cientist will have to learn how to turn on specific genes when a protein of enzyme is low, and off to avoid too much to ensure the delicate chemical makeup of our bodies is not disrupted. (Blachford, 467) Although there are many risks of gene therapy, the benefits remain to enticing to ignore. Gene therapy poses many risks, but may prove the ideal solution for countless diseases. As seen throughout the past few decades, gene therapy can cure genetically inherited diseases by introducing therapeutic genes into the body. 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.
Once that is done the scientist would then introduce the protein into human cells. Gene therapy can be simply viewed as inserting bits of foreign DNA into a patient’s tissue in hope... ... middle of paper ... ... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today.
Viruses (that have been modified to not cause harm) are well known vectors for gene therapy. Retroviruses and adenoviruses are commonly used viruses that are used for gene therapy; they may or may not incorporate their own genes into the gene being delivered to the human chromosome. The vector could be injected by an IV into a specific part of the body in which, it can be taken up by individual cell (invivo). Another option could be to take a cell out of the person’s body and in a laboratory place the vector in the cell (exvivo). Later the ce... ... middle of paper ... ...efore we know it doctors will be prescribing genes instead of pills or painful injections.
With the kind of technology that is available to doctors and scientists, treatments and cures will begin to pop up as we continue to advance in technology. Genetic engineering is going to be the topic to look out for. With the incredible discoveries done from modifying genes, it has resulted to millions of lives saved. Vaccines had come a long way from the first discovery, inching towards the one hundred percent success rate. Fredrick made a ground breaking discovery with diabetes and provided a treatment and saved millions.
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.
A single alteration to this code can result in a lethal disease. In gene therapy, genes with the cor rect code are inserted into the cell. The method of choice to insert desired genes into human cells is through a virus vector in which the virus inserts modified DNA to replace the DNA that is causing the genetic problem (Coutts). Once the DNA is in th e cell, it can code for the desired protein needed to make the patient well. If the cells that are treated in your body are not reproductive cells, this type of gene therapy is called somatic cell therapy.
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).