What Is Gene Therapy?

There are two main groups of gene therapy and they are germ line gene therapy and somatic gene therapy (Baksh, 2007). Germ line therapy consists of germ cells being modified by the introduction of functional genes, which are ordinarily integrated into their genomes (Garbutt et al., 2011). DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line

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

     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.

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.

Improvements with treatments and screenings, those affected with cystic fibrosis now live well into their 20s and 30s, and some are living to their 40s and 50s.

Cystic fibrosis is a rare life threatening disease, that as of now has no cure. But with help from extensive research that is being conducted, there is soon to be a cure in the future for this disease. The disease attacks the digestive system and slowly over time shuts down the lungs.

Cystic fibrosis (CF) is an inherited autosomal recessive disorder that affects the lungs and digestive system most often. In the United States some 30,000 children and adults have CF. There are approximately 1,000 new cases of cystic fibrosis diagnosed each year in the US with 70% of patients diagnosed with CF by the age of two, 40% of patients with CF are 18 or older. In the 1950's most children with CF did not survive to attend elementary school, but in 2006 the median age of survival was 37 years (Cystic Fibrosis Foundation, 2007).

Most of the people diagnosed with CF are usually in the age of 2. The predicted age most people with CF is at age 40. 97-98% of men with CF can not have children because of congenital bilateral absence of the vas deferens. Most of the people with CF, there parents had to be carriers and they gave their children the disease. For example if 2 carriers had a baby the chances of the baby having CF is, ¼ will not have CF, ½ will be a carrier, ¼ will not be carriers or infected. Symptoms of CF vary with each person. Some usually the symptoms are coughing, lung infections, inability to gain weight and, fatty stools. Lung infections are when the bronchial tube gets inflated. It is really rare to get CF it can either last 1 year or it can be for life. If you would want to know if you have it, it requires a medical

In the United States there are close to thirty - thousand people and seventy thousand people worldwide living with Cystic Fibrosis today, with the majority of them being over the age of eighteen. CF has over eighteen-thousand mutations, but most of the tests can’t always pick up on them, only the more common mutations of CF. By the age of two, over seventy-five percent of kids containing CF are diagnosed, making over one-thousand new cases of Cystic Fibrosis diagnosed every year. Cystic Fibrosis is caused when both the parents of the child have the CF genes which is why it is a recessive genetic disorder. Thus making it something that isn’t

Gene therapy: Is a treatment method that involves the manipulation of an individual's genetic makeup. A form of therapy that attempts to fix the defective gene which is causing the disease.

There is no cure for cystic fibrosis, but medication can be provided to help ease the symptoms that accompany the disease. Things like antibiotics can treat and prevent lung infections. A person can also take mucus thinning drugs that help to improve lung functions, by helping you cough out the mucus. Furthermore, a person can also take in pancreatic enzymes that help to digest food in the small intestines and stomach. If a person’s intestine folds in on itself, then the person would have to have surgery that removes the part of the intestine. If the blockages in the ducts in the lungs become resistant to medication, or antibiotics, then the person might need to have a lung transplant. Cystic fibrosis does not occur in the set of lungs that have been transplanted into

I have been dealing with cystic fibrosis my entire life because my fourteen year old brother was diagnosed with it when he was born. He was in

The severity of this disease differs over time, as time goes on, symptoms can get worse. As a child the lung functions typically declines and over time people end up experiencing sever breathing problems. Respiratory is the leading cause of death in people with CF. As time goes on and treatments and advancements in research and care improve, as do lives of people with CF. The general life expectancy of someone with CF is 41 years, but some people can live to make it to their 50’s or even longer. Back in the 1950’s people with CF had a hard time living to attend elementary school. Early treatment of this disease can help the length and quality of life. With that being said, there is still no cure.

(cure, care, prevention, if any?) There is currently no cure for Cystic Fibrosis. A person who has Cystic Fibrosis may consume up to 40 capsules daily which assist them to digest food, in some cases sufferers always may need to do up to three hours of airway clearance every day. Treatment generally includes some of the following: intensive daily physiotherapy to clear the lungs, salt and vitamin supplements, exercise (as it is important to clear airways) and antibiotic therapy to treat lung infections. From birth a person who has Cystic Fibrosis undergoes consistent medical treatment and

In short, gene therapy is a method in which DNA Is used to fix a genetic problem. These problems can include missing or genes that don’t work. The right genes are delivered to the cell via harmless viruses for their provirus properties. The viruses then insert the good copy of the gene into the patients DNA where the cells machinery reads the information like a manual and makes RNA proteins so when it divides, ideally, the problem will be fixed. There are two types f gene therapy: Germline (targets egg/sperm cells; stops disease in its tracks) and somatic (targets somatic cells; after the disease is active).The methods for gene therapy that are being tested include: