Gene Therapy For Cystic Fibrosis

Cystic fibrosis is an genetic disease that can cause bad damage to the lungs and other organs in the body. It is a common genetic (Gene) disease affecting to geriatric, (adults) young adults, or and kids. Now they’re still searching for a cure, but the only thing that is currently available is a donor’s lungs.

In infants symptoms include coughing, wheezing, excess mucus in the lungs, shortness of breath, extremely salty skin and decelerated growth. With infants it is very common for them to develop pneumonia infections because of all the bacteria that resides in the lungs. With treatment, most patients with cystic fibrosis live into their 20’s and 30’s, some individuals with milder cases can live longer. Death is most often due to end-stage lung disease.Thus far no cure for the disease has been found. Although, research on gene therapy is promising. Treatment is generally aimed at alleviating symptoms, preventing infections, and slowing the progress of the disorder. (Egan’s 10th Ed,

The Cystic Fibrosis Foundation provides support to help improve the lives of people who live with the disease. The foundation’s main goal is to find a cure to Cystic Fibrosis is an inherited disease caused by changed in a gene on chromosome seven. It is described as a buildup of mucus in the lungs and organs. The mucus in the lungs clogs the airways and bacteria grows, gets blocked in and eventually leads to infections that causes lung damage. After lung damage has occurred, respiratory failure then happens.

Every day, people die from Cystic Fibrosis. That is why Cystic Fibrosis is known as the most deadly inherited disease. There is no real cure of CF but, right now there are many ways to ease your symptoms and help you manage the condition. “Every day, people with CF complete a combination of the following therapies Airway clearance, Inhaled medicines, Pancreatic enzyme supplement and sometime oxygen therapy depending on how severe your condition is”(McLoud). Since there is so many new advancements in treatment, people who have CF now live twice as long as they did 30 years ago.

CF is an autosomal recessive disorder that is predominately found in European decent. It affects various systems in the human body, however, it is the respiratory system that contributes to the high mortality rate due to pulmonary decline. This is due to a mutation in the CFTR gene, depending on what mutational class it falls under can indicate the severity of their clinical outcomes. The lack or complete absence of functionality of the CFTR gene results in mucus accumulation in the airways, which consequently makes them more prone to infections that may hasten their lung deterioration and even endanger their lives. Even though there is no cure for CF a number therapeutic classes are implemented usually in concomitant with each other to delay progression of lung disease and provide symptomatic relief.

Cystic Fibrosis (CF) is a disease that causes problems within the lungs and digestive systems (pancreas, livers, and intestines). It occurs due to the abnormal transport of chloride and sodium across an epithelium, leading to excessive secretion of thick mucus in these two major areas of the body. This makes breathing difficult as the thick, sticky mucus clogs the airway, and it can also result in sinus infections, poor growth, infertility, and reduced life expectancy. However, these can sometimes be treated with antibiotics and other medication, like medicated inhalers.’ It is an autosomal recessive genetic disorder and is caused by one of many different mutations in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR)’ . Because CF is a genetic disorder it is able to be inherited therefore can undergo human manipulations such as Selective Breeding and Gene Therapy.

     Cystic fibrosis is an inherited autosomal recessive disease that exerts its main effects on the digestive system and the lungs. This disease is the most common lethal genetic disorder in Caucasians, affecting one out of 2,500. On the bioethical front, CF was the first human genetic disease to be cloned by geneticists. The intent of this paper is to describe how the cystic fibrosis gene was identified, how the

Research on CFRD is critical in the care of the CF patient because it cannot be treated like typical diabetes. The pathophysiology, diagnosis,

Cystic fibrosis is a life-limiting disorder which causes sticky secretions in the lungs and in the ducts of the pancreas. Children born with this disorder face a lifetime of struggling to breathe, and treatments consisting of percussion on the back to loosen the mucous. In addition, these children develop diabetes as they get older. There is very little quality of life for these youngsters. The discovery of F508del in the 90s has helped to change that outlook. This is the transmembrane conductance regulator gene (CFTR) which was found to be the most common cause of cystic fibrosis. It functions as a chloride channel regulated by cyclic AMP dependent phosphorylation. Almost 2,000 variants of this gene have been reported, 40% are predicted to cause substitution of a single amino acid, and 36% are expected to alter RNA processing. This has led to research into the possibility of genetic modifiers, which if successful could either improve the severity of, or eliminate this disorder. The CFTR gene in mice has been successfully manipulated to derive lines that do not expresses CFTR, and lines that express CFTR bearing variants equivalent to those expressed in humans. The absence of lung disease similar to that seen in humans with cystic fibrosis has been ascribed to the presence of alternative pathways for chloride transport in mouse epithelial cells.

At a simplistic level, gene therapy is the use of one’s own cells to provide a therapeutic effect. In the majority

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,

Cystic fibrosis is one of the major diseases that have no known cure but is prohibited by genetic engineering. Cystic fibrosis is a disorder in which attacks lungs and the digestive system. Before genetic engineering was discovered by scientists, people of that generation were incapable of having their genes modified. For those who are diagnosed with cystic fibrosis, they are given medicine to ease the pain. When Cystic Fibrosis is not properly attended to it can lead to diabetes or even lung cancer. Although genetic engineering has the possibility of saving a human beings' life, it also has the possibility of making it worse and causing

Cystic fibrosis. We may be able to produce effective genetic therapy against cystic fibrosis. Ian Wilmut and colleagues are already working on this problem.

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.

“Helper-dependent adenoviral vectors (HDAds) possess long homology arms that mediate high-efficiency gene editing. These long homology arms may permit simultaneous introduction of multiple modifications into a large genomic region or may permit a single HDAd to correct many different individual mutations spread widely across a gene” says Palmer. “HDAds can efficiently deliver foreign DNA into the nucleus of target cells. Furthermore, they are deleted of all viral-coding sequences, thereby reducing their toxicity and increasing their cloning capacity to 37 kb.” Twelve 2 bp insertions were made into an HDAd by this team of scientists, in order to determine how much of the modified gene-of-interest had been introduced into the CFTR locus after homologous-recombination. Of the 83 targeted recombinants which were analyzed, the data revealed that even markers a good distance from the desired marker (11 kb, the farthest being 22 kb) were introduced to the target gene efficiently and at high frequencies (about 21.7%). The results of this experiment are consistent with the idea that HDAds can make changes over large genomic regions, and may prove useful in the advancement of individual treatment for genetic disorders such as Cystic Fibrosis.