Cystic Fibrosis - Fred Hutchinson Cancer Research Center

Diseases / Research

Cystic Fibrosis

Fred Hutch researchers are pioneering new ways to correct the defective genes in cystic fibrosis patients, laying the foundation for future new therapies. 

A Hutch scientist played a key role in the success of gene therapy tools making it possible to transfer genes into specific cells tha could make cure a possibility.

Fast Facts

  • 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. Mucus can also 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.

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Treatment & Prognosis

Pursuing new genetic treatments – Fred Hutch researchers developed new approaches to treating lung disease associated with cystic fibrosis and alpha-1 antitrypsin deficiency. Dr. Miller, who recently retired, was one of the first researchers to efficiently transfer genes into human cells, a breakthrough that is providing scientists worldwide with new gene therapy tools.

Research focused on developing more efficient viral transport vehicles, or vectors, for human gene therapy. Miller and his collaborators made significant improvements in gene transfer to blood cells and immune system 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.

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