Cystic Fibrosis Disadvantages

for Cystic Fibrosis
Cystic Fibrosis, also called CF, is the most common life threatening disorder in the US. It is an inherited disease of the secretory glands (National Blood, Heart and Lung Institute, 2013). Affecting about 30,000 people, it is an autosomal recessive disorder due to mutations in the CF transmembrane conductance regulator (CFTR) gene (National Blood, Heart and Lung Institute, 2013). Usually diagnosed at a young age, CF causes the mucus, sweat and digestive fluids to become thick and sticky (Mayo Clinic) resulting in ducts, passageways, and tubes in the lungs and pancreas becoming plugged, causing respiratory, digestive and pancreatic problems. As of now, this disorder cannot be cured, but it can be treated. With the field
One is that there is hope for a cure. While many trials and test have been run, because gene therapy can remove the defective gene, there is a possibility that it can cure CF. Gene therapy also provides a large scale of treatment (NLCATP, 2014). Since CF affects the respiratory system like many other diseases, gene therapy can wipe out the “abnormal” gene in carriers so it will not be inherited by later generations.
Disadvantages of Using Gene Therapy A disadvantage to using gene therapy is that there is a small amount of knowledge on it. A large amount of people does not understand how gene therapy fully works or the safety issues it causes. Another con is that doctors do not know how long gene therapy will be able to work. Clinical trial results are only short term and show a small amount of benefits (NLCATP, 2014). A moral drawback is that religious groups feel as if it shouldn’t be used because it changes your genetic makeup. With more research, these disadvantages shouldn’t pose a problem in the future of gene therapy and the cure for Cystic Fibrosis.
Clinical
However, a cure could be found through gene therapy. Diagnosed at a young age, signs and symptoms vary between each person, and treatment can ease complications. Gene therapy can take out the abnormal gene and replace it with a normal copies of the CFTR gene to rid the patient and carrier of the mutation. Clinical trial results show that gene therapy works well in showing small improvements in lung infections and inflammation caused by CF. Although these are small improvements, they can become better with more research and better vectors for delivery. Gene therapy needs to improve entrance into the linings of the lungs but can work well with current routes. With wave one and two of the UK Cystic Fibrosis Gene Therapy Consortium research rapidly progressing and going well, scientists are able to choose a form and amount based on results of their current phase 2b trial and formulate their next trial hoping for more