Cystic Fibrosis

• Cystic fibrosis is a genetic disease that can cause the body to produce unusually thick mucus, affecting the lungs, pancreas, liver, intestines, sinuses and sex organs.
  
  
• Cystic fibrosis produces symptoms of varying type and severity. Some patients produce abnormally thick, sticky mucus in their airways, leading to breathing problems and bacterial growth that can cause hard-to-treat infections. In addition, mucus can obstruct the pancreas, preventing enzymes needed to help digest food from reaching the intestines.
  
  
• More than 10 million people carry the defective gene responsible for cystic fibrosis but have no symptoms. To develop the disease, an individual must inherit a defective copy of the cystic fibrosis gene from each parent.
  
  
• There is no cure for cystic fibrosis. In the 1950s, few children with cystic fibrosis lived long enough to reach school age. Thanks to advances such as those made at the Hutchinson Center, many patients are living well into adulthood.

Gene Therapy as treatment – One of the first researchers to transfer genes efficiently into human cells was Dr. Dusty Miller. This breakthrough continues to provide scientists around the world with the tools to develop and extend knowledge into gene therapy.

Research in Miller's laboratory focuses on the basic biology of two types of viruses, with the goal of developing more efficient viral transport vehicles, or vectors, for human gene therapy. For Miller and colleagues, one major objective is to develop treatments for lung disease associated with cystic fibrosis and alpha-1 antitrypsin deficiency.

Collaborations with Drs. Hans-Peter Kiem and Robert Andrews have led to significant improvements in gene transfer to blood cells and cells of the immune system. They have employed vectors based on an animal virus that uses a receptor found on the surface of blood-making stem cells. The ability to transfer genes into such cells is critical to achieving the long-term integration of curative genes for a variety of diseases, including genetic diseases and certain cancers.