To understand what gene therapy cystic fibrosis is, first break down the terms. The term cystic fibrosis refers to a hereditary disorder. The symptoms of this disease is caused by the mucus membranes production of thick mucus as opposed to a thinner, slippery mucus. This mucus builds ups and causes the pancreas to malfunction. Important enzymes no longer reach the digestive system, and as a result various parts of the body begin to breakdown. Lung function, loss of bone density, malnutrition, raised blood pressure and constipation are major symptoms for those with cystic fibrosis. The use of antibiotics, painkillers, respiratory medicines and supplementary vitamins can be used for minor relief. Various experimental gene therapy for cystic fibrosis treatments have been tried, but nothing has yet cured the disease.

Gene therapy is an experimental medical treatment which involves trading damaged genes with functioning genes. This is sometimes done by a simple injection into any part of the body, but more advanced methods have been developing in laboratories worldwide. There have been major breakthroughs involving gene therapy, but because the use of stem cells are closely related there has been a lot of controversy surround the subject.

The most popular treatment for cystic fibrosis using gene therapy is the use of a method involving mutating viruses. The basic principal of how a virus works is that it is an invading foreign body which seeks a host cell in order to replicate more viruses. Viruses are very basic and contain a few enzymes which carry its genetic code. Scientists are trying to create viruses which carry the enzyme which will replicate the missing or damaged gene. While this method could very well cure cystic fibrosis, it is not without considerable risks. Viruses are dangerous, and for a person with a healthy immune system it is sometimes impossible for it to fight it off. In the case of gene therapy for cystic fibrosis that involves the use of viruses, if a mistake were made and the virus was not properly developed, a potentially fatal result could occur. Most people with cystic fibrosis have severely weakened immune systems, so even a helpful virus could be dangerous. Scientists will consider to experiment on this method in an attempt to develop a cure.