Essay on Cystic Fibrosis and Gene Therapy

Nearly 1 in every 30 Americans are a carrier of the recessive gene that leads to CF (About Cystic Fibrosis, n.d.). Although CF is a life-threatening condition, through the advancement of treatments and care, there has been a steady increase in life expectancy and improvement in the quality of life. The current mean age of survival is now 40. Although that does not seem very high, sixty years ago, a child diagnosed with CF did not survive childhood (About Cystic Fibrosis, n.d.). CF is a complex disease, where the types and severity of symptoms can differ widely from person to person. Symptoms may include fatigue, salty-tasting skin, persistent cough with phlegm, wheezing and shortness of breath, lung infections, and poor growth and weight loss (Cystic Fibrosis Symptoms, Causes & Risk Factors, 2018). Many different factors, such as the age of diagnosis, can affect an individual's health and the course of the

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,

     Today, there is no cure for Cystic Fibrosis, but if the disease is found in an individual early enough, the patient can be recovered. About 85 to 90% of patients will have problems absorbing nutrients from their intestines. And because enzymes are lost when diagnosed, enzyme supplements are required to prevent bad nutrition and malnutrition. There are two main body parts that can be cared for the treatment of Cystic Fibrosis: The lungs and the chest. Lung problems can also be treated, but not cured. Antibiotics and other drugs may loosen the thick mucus in the lungs, but if it fails, a transplant lung may help to extend life. But about four hundred people die each year while waiting for a lung transplant. Also Chest physical therapy is a technique done by vigorous claps on the back and chest to loosen the thick mucus from the lungs. The average lifespan of an individual with CF is about thirty years.

“About 1 in 4,000 children in the United States are born with CF” (Crosta, 2009) and “one in every 3,600 children born in Canada [have] cystic fibrosis” ("Cystic fibrosis Canada," 2011). According to several resources a consensus has formed which proposes that people with northern European ancestry, specifically those that are white, are more likely to be carriers of the disease and thus more at risk of having children with the mutated gene. This risk is even more likely if the other parent of the child is a carrier. Another risk factor for CF is “family history, since the disease is hereditary it increases they likely hood of

Cystic Fibrosis, a very serious inherited genetic disease, is also known as CF and sixty-five roses. This disease affects one in every 3,000 live births. It may first appear in a newborn, but can appear all the way up until a young adult. However, ten percent of most cases are apparent at birth. CF affects the lungs and causes a build-up of abnormally thick mucus which leads to chest infections, and CF also affects the reproductive system. Doctors do not know what causes the mucus to thicken. CF’s infections usually lead to death in childhood and early adulthood. Most people infected with CF had a life span into their teens long ago. Now, due to advanced technology, the life span is in the fifties or older.

Can you imagine struggling and gasping for air for an everyday normal? It all ended for her in the year of 1988. More than 30,000 Americans are diagnosed with it a year. Leslie Adelynn Lacey, a young girl from the small town of Kosciusko, Mississippi, was diagnosed with cystic fibrosis when she was only six months old. Cystic Fibrosis, (CF) is an inherited life-threatening disorder that damages the lungs and digestive system. Mucus builds up in the lungs and blocks the person’s airway, causing this person to have trouble breathing regularly. CF is a genetic disease, meaning that a person does not catch it, it is passed down through DNA. Cystic Fibrosis last with a person his or her entire life time. It occurs throughout the world in every race and ethnicity. There is absolutely no way to prevent a person from contracting CF. Lifespan expectancy is usually 21 for a patient diagnosed. Even though there is no cure for cystic fibrosis, the advancements in learning about the condition, genetics and therapy are a working progress throughout medical experts, and maybe, one day there will be a cure.

Genetic diseases are the results of changes in an individual's DNA, usually inherited. An example of a fatal genetic disease is cystic fibrosis (also known as CF). This is the most common fatal genetic disease in the United States, over 30,000 people in the U.S. have cystic fibrosis and about 1,000 people are diagnosed each year. This disease attacks many different parts of the body, but the most affected area is the lungs. Cystic Fibrosis is genetic, has obvious symptoms, and is deadly.

Cystic fibrosis is the most common lethal genetic disease that affects children and young adults. The disease is inherited and mainly affects the lungs and the digestive system. It is estimated that there is one case of cystic fibrosis in the country in every 3,600 children. More than 4,100 children and young adults suffering from cystic fibrosis attend specialized cystic fibrosis clinics (Horsley, Cunningham, & Innes, 2015).

Cystic Fibrosis is a recessive disorder that is a life threatening disease that causes persistent lung infections and progressively limits the ability to breath. Unfortunately, there is no cure for this genetic disorder, but Airway Clearance, Inhaled Medicines, and Pancreatic enzyme supplements can increase the person’s lifespan incredibly. There are about 70,000 people worldwide with this disease. Approximately 1,000 new cases of cystic fibrosis are diagnosed each year. The average human with cystic fibrosis lives to about 30 if they take their medicine.

Cystic fibrosis is a scary disease. The life expectance for people with cystic fibrosis is in batween thirties and forties. It was discovered in 1938. There are more than 1,500 mutations that have identified in the single gene.

Cystic Fibrosis is a genetic disease that has been found to be life threatening. It causes buildup of mucus in the lungs and in other organisms. In the lungs, mucus can get so built up that it blocks the airway and can lead to infection and possibly lung failure.

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

While cystic fibrosis (CF) is not a new disease, there is still a lot to learn about it. In 1938 a pathologist, Dr Dorothy Andersen, provided the first clear description of cystic fibrosis. Before this time there had been reports of people that had the symptoms of someone with CF. During the seventeenth century children with the symptoms of CF were thought to be bewitched and their life expectancy was very short. Dr Dorothy Andersen gave this disease its name because cystic fibrosis refers to the scarring that is found on the pancreas. People with CF also have associated diseases like salt-loss syndrome, obstructive azoospermia, and gastrointestinal abnormalities. CF is inherited from one’s parents, making it a genetic disease. CF is

The impacts of CF on a human can be very serious and life-shortening. A person with CF usually has their airways blocked by the mucus produced, therefore having trouble breathing. This can also result in the person running out of breath easily, and not being able to exercise for long periods of time. The person affected by CF needs to be checked regularly at Cystic Fibrosis Care Centers to maintain their CF. They should always take the medicine prescribed by their CF manager/doctor. A person affected by CF can’t do all the regular activities that people who aren’t affected by CF can do, like running, going to the gym and general strenuous activities. People affected with CF usually have a life expectancy of around 40 years assuming that the

Will it be cured? What are the existing treatments? Cystic fibrosis is known as the most lethal genetic disease of Caucasians (O’Sullivan and Freedman, 2009). In the general population, cystic fibrosis has an incidence of 1:1600 and a carrier frequency of 1:25 (Kreindler, 2010). The good news is the median survival of cystic fibrosis patient has risen from only less than a few years to more than 37 years in the past 70 years (Kreindler, 2010). The use of therapies such as Pancreatic enzyme replacement therapy (PERT), Gene therapy and Novel CFTR-directed therapies helps achieve the remarkable success (Oates and AMOS,