What Are The Pros And Cons Of Gene Therapy

By using somatic gene therapy, it is possible to improve the pulmonary disease in cystic fibrosis. Somatic cell gene therapy only affects body cells and does not effect the sperm and egg cells, as it does in germ line therapy, this therefore means that will not prevent the disease occurring in future generations. Although this type of gene therapy has be seen to work and increase the life span of a person suffering from cystic fibrosis, it has to be done several times over the patients life because the effects do not last very

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

Cystic fibrosis is known to be one of the most common and deadly diseases in Caucasians, affecting 1 in 2500 children. This percentage results in 30,000 individuals within the United States to be diagnosed with CF. There are over 1900 mutations of this gene that cause a wide variety of severities within this disease. (McCance, Huether, Brashers, & Rote, 2010) Due to its complex mutation and unknown cause, only treating the symptoms of CF have been the main treatment protocol to this disease. Current treatments are cumbersome and expensive providing patients with life expectancy only into their twenties, but usually younger in most cases. There has been specific progress towards a cure involving gene therapy providing hope for a cure to

“A team of scientists conducted a trial therapy that replaced defective genes that cause cystic fibrosis. The results showed noteworthy advantage that enhanced patients' lung function. This innovation was developed by the technology firm Imperial Innovations. The worn-out genes were replaced by using inhaled molecules of DNA, which send normal working duplicate of the gene to lung cells. "Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group," said Eric Alton, the team's lead researcher, who works at Imperial College London.”

Gene therapy is another approach to eradicating genetic diseases. “Gene therapy was considered for a long time appropriate only to treat monogenic diseases by means of a therapeutic gene transfer for replacement of a defective homologous gene.” (NPR 1) It is a very efficient way to prevent genetic diseases such as Crohn's. Modern day technology and scientists are curing many genetic diseases around the world using many forms or genetic

Once this is determined, we will use gene therapy to treat Cystic Fibrosis.  We will use the adenovirus treatment mentioned above, but in order to counter the fact that it doesn’t last long enough, we will propose more frequent dosages of this treatment.  The dosages will be administered with aerosol.  We will have to do research to find the right amount of time to wait in between dosages because if we give them too close together, this will cause too much inflammation.  If this approach does not end up working, we will also consider finding a way to enable the adenovirus once it has been administered.  This would allow the adenovirus to replicate and stay in the patient’s DNA, which could possibly cure CF.  Another option is finding a way to lessen the cell-mediated immune response.  Once we performed research and clinical trials, we would determine which combinations of these options would work to cure Cystic

Gene therapy is one of the newest, yet controversial solutions to treating many diseases, including Cystic Fibrosis. Gene therapy is the insertion of a normal gene into deficient cells using an appropriate vector (Hart, et al. 2006). However, it does have potential risks, leading to the argument of if they should be used or not, and if used, is a viral or non-viral approach better. It becomes complicated since many symptoms of these diseases begin in early childhood, and deciding whether or not to subject a child to these possible dangers is a hard choice, but do the benefits outweigh the risks? As stated above, Cystic Fibrosis can lead to early death, around 37 years old (Hulslander 2017), so if death is the impending result, shouldn’t every possible measure be examined, if not explored?

Gene therapy is the insertion of normal or genetically altered genes into cells to replace defective genes. The gene is usually inserted into

There have been multiple setbacks in this technology. Some immediate side effects is that the body thinks that the virus inserting the healthy gene is a virus and tries to fight it. A severe response to the virus could lead to the death of the individual. Another major side effect in the long run is the development of other diseases. There have been multiple reports of operations that have gone successful, but in a few years that patient developing diseases like leukemia and most of the patients died. Right now there are probably more negative effects to gene therapy and more research would need to be developed to ensure that the likelihood of death is not so high. More research and operations on animals would have to be done to ensure it is safe on

Over 70,000 people worldwide have cystic fibrosis, a genetic disease that causes a buildup in mucus, difficulty breathing, and damage to the lungs (Cystic Fibrosis, n.d.). Currently, there is no cure, but treatments such as gene therapy may help those who have the disease (Gene Therapy, n.d.). Gene therapy is a method of treating genetic diseases in which a vector such as a virus delivers the genes to target cells. This causes the expression of a person’s genes to change (Hanna, 2006). Somatic cells or germline cells can be treated (Gene Therapy, n.d.). Gene transfer to somatic cells changes the recipient’s genome (Hanna, 2006). Somatic gene therapy is likely to be a successful way to treat diseases caused by a single mutation (Gene Therapy,

Gene therapy is term that offers a wide range of treatment types. These types of

Gene therapy is an experimental technique that uses genes to treat or prevent disease. Currently, there are a lot of studies and experiments that allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or direct surgery. Researchers are testing several approaches to gene therapy, such as replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating, or “knocking out,” a mutated gene that is functioning improperly, introducing a new gene into the body to help fight a disease, and helping to regenerate precious cells. Although gene therapy has a very active and promising approach for a number of disease, there are still several risks and side effects. Hence, the therapy

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

Cystic Fibrosis (CF), is another disease that is taking to genetic therapy. If a corrected gene could somehow enter the cells that line the lungs, it will then start producing the critical proteins that CF patients need. This has been done, although in small quantities. These results, however, have raised hopes that sometime in the future, CF may be curable.

Gene therapy is the method of use of genes by inserting a functioning gene into body cells in order to correct the disease by inactivating altered genes with inserting a genetically modified gene into the body in place of drugs to fight disease (Verma, 1990).