Cystic Fibrosis and Gene Therapy

A Learning Experience

Gene Therapy

Gene therapy is an experimental and controversial treatment that uses genes to treat or prevent disease. This includes:


  • Replacing a mutated gene that causes disease with a healthy one
  • Inactivating a mutated gene that is functioning improperly
  • Introducing a new gene into a body to help fight disease, like cystic fibrosis

Cystic Fibrosis and Gene Therapy

Cystic Fibrosis is a genetic mutation that causes the lungs to fill up with a thick mucus. Many people with this disorder have a life expectancy of 35 years or less. A new drug called Ivacaftor is helping to increase lung function. Gene therapy is helping in Cystic Fibrosis by activating the inactive cell. There are two forms of gene therapy used to treat Cystic Fibrosis. The first is germ line gene therapy, and this form not only helps the person being treated but also helps those peoples children and so on. The other form is called somatic gene therapy. This type involves changing the defective gene in the individual but the change won't be passed to the next generation.

Related Jobs and Professions

Why These are Related

Jobs and Professions

Biotechnologist can use the systems in the body to find out how Cystic Fibrosis and Gene Therapy react together throughout the body. Pathologist can help to study the tissues of the lungs in patients where Cystic Fibrosis has occurred. Not only will they be able to study the tissues of the lungs but they will also help to study the tissues effected by the Gene Therapy treatment. Geneticist help to find the way that Cystic Fibrosis can be passed down genetically through generations.