Gene Therapy
What is it?
Gene therapy research exemplifies a primary use of genetic engineering in the medical field. By definition, gene therapy is the insertion of selected genes into a patient’s cells in place of surgery or drug therapy in order to “fix” defective genes responsible for disease development. Though this type of therapy cannot yet be implemented in regular medical practice due to the uncertainty of possible side effects, it provides a promising alternative to invasive surgeries or costly medicines. How does gene therapy work? According research conducted by the Human Genome Project, there are several ways to replace or repair damaged genes using gene therapy: 1. Nonfunctional genes may be replaced by inserting functional genes into a nonspecific location. 2. Normal genes can be used to replace abnormal genes using a process called homologous recombination. 3. A faulty gene can undergo selective reverse mutation to restore the gene’s original function. 4. Gene expression could be regulated using gene switches, which would control the behavior and development of certain cells and tissues in the human body. Though each of the above methods may provide the desired results, the most common method explored in gene therapy research involves the replacement of disease-causing genes with normal DNA sequences. Undamaged DNA is crucial to the normal growth and function of tissues because our DNA provides the “codes” our cells use to build proteins. Proteins are responsible for a wide variety of tasks in the human body, such as structure, transport of nutrients and wastes, and muscle movement. When a person has cells with damaged DNA, then the body may no longer function properly, leading to disease. However, functional DNA cannot be inserted directly into a patient’s cell without some sort of carrier molecule, or vector. Viruses provide the most common type of vector because of their ability to integrate viral DNA into host DNA. Scientists have developed a way to manipulate specific viruses to carry “viral” human DNA, which the virus will then integrate into the host cell’s DNA. If successful, the host cell will then begin producing the proper proteins. |
What type of research is there? According to the American Medical Association (AMA), gene therapy treatments are most successful when used to treat diseases caused be defects in single-genes, such as adenosine deaminase (ADA) deficiency. The treatment of a child afflicted by ADA in 1990 represents the first gene therapy clinical trial. Researchers are also investigating diseases such as selected types of cancer, AIDS, Alzheimer’s disease, cystic fibrosis, and many others. The AMA reports that the only disease that has actually been cured using gene therapy is severe combined immunodeficiency disease (SCID), which is a disease in which a person’s blood does not produce enough white blood cells. This disease is often fatal during early childhood, or even soon after birth, because the immune system is unable to fight infections. However, if detected early enough, children affected by this disease could receive gene therapy in order to boost the blood’s white blood cell count to normal levels. The Boston Children’s Hospital’s pediatric health blog provides an exciting example of how gene therapy saved the life of a baby boy affected by SCID. |
Why should gene therapy research be funded?
Though many people are often apprehensive about the social and economic effects associated with funding medical research such as gene therapy research, DELTAFORCE1 believes that this research must be funded. Gene therapy provides an effective, less invasive alternative to many procedures commonly used to treat diseases caused by gene defects. Because the genes used in this type of therapy come from the patients’ own stem cells, there is no donor waiting list for transplants, and risk for treatment rejection (caused by the immune system) is greatly reduced. Furthermore, gene therapy could provide patients with a cure for diseases that are fatal, or detrimental to the quality of a patient’s life. Because it is rather costly to conduct gene therapy research, many gene therapy institutes must receive funds from outside donations and sponsors. Likewise, hospitals that treat diseases that have already been proven to respond to gene therapy also greatly need funds to provide families with this treatment.
Though many people are often apprehensive about the social and economic effects associated with funding medical research such as gene therapy research, DELTAFORCE1 believes that this research must be funded. Gene therapy provides an effective, less invasive alternative to many procedures commonly used to treat diseases caused by gene defects. Because the genes used in this type of therapy come from the patients’ own stem cells, there is no donor waiting list for transplants, and risk for treatment rejection (caused by the immune system) is greatly reduced. Furthermore, gene therapy could provide patients with a cure for diseases that are fatal, or detrimental to the quality of a patient’s life. Because it is rather costly to conduct gene therapy research, many gene therapy institutes must receive funds from outside donations and sponsors. Likewise, hospitals that treat diseases that have already been proven to respond to gene therapy also greatly need funds to provide families with this treatment.