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Cystic Fibrosis

With funding from the American Lung Association, researchers at the Wake Forest Institute for Regenerative Medicine (WFIRM) are investigating a potential new therapy for cystic fibrosis (CF).

About 1,000 new cases of CF are diagnosed each year. People with the disease have a gene defect that causes the body to produce thick, sticky mucus that builds up in the lungs and digestive tract. The mucus can lead to life-threatening lung infections and disrupt the normal breakdown and absorption of food. According to the Cystic Fibrosis Foundation, the median life expectancy for people with the disease is the late 30s.While the genetic cause of the disease has been known for more than 20 years, a curative treatment has not yet been developed.

A potential therapy for the disease may be a type of stem cell found in placenta (afterbirth) and amniotic fluid that was discovered by researchers at WFIRM. These cells have been shown to have the ability to form normal lung cells and to produce lung-specific genes and proteins, including the gene that is defective in CF patients.

The scientists will first explore the use of these cells as an injectable therapy in animals with a CF-like disease. MRI will be used to track the location of the cells. The ultimate goal is to develop a cell replacement therapy to restore normal gene function in patients with CF.

 

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Our researchers are trying several different strategies to restore the body's natural capacity to produce insulin.

 

 

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