GENE THERAPY VECTOR

CLINICAL CENTER GENOTYPING CENTER
Information regarding Cystic Fibrosis, the types of research being performed to finds its cure.  Learn more about gene therapy, and treatments for Cystic Fibrosis as well as volunteering opportunities.
The Center has many research laboratories dedicated to finding a cure for Cystic Fibrosis.  Meet the different lab directors, their lab members and the laboratories particular focus.
A complete list of publications from the Centers lab directors that are related to Cystic Fibrosis.
Apply for open positions at the Cystic Fibrosis Research Development Center.
Visit other websites of organizations and associations affiliated with Cystic Fibrosis.
Letter from the Director addressing the Centers continued plan for the future.
 
 
 
 

 

 

 

 

 

 

 

 

 

 

 

 

   
 

 

   

 

Cystic Fibrosis Treatments

Since the defective CF gene was discovered in 1989, the pace of CF research has greatly accelerated.  In 1990, scientists successfully made copies of the normal gene, and added them to CF cells in laboratory dishes, which corrected the defective cells.  The next major step was achieved in early 1993 when the first experimental gene therapy treatment was given to a patient with CF Researchers modified a common cold virus to act as a delivery vehicle - carrying the genes to the CF cells in the airways.  Several Foundation-supported studies are underway to test new versions of the cold virus, and other cutting-edge technology, such as fat capsules (liposomes) and synthetic vectors.  For more information on CF gene therapy, see Gene Therapy and Cystic Fibrosis. 

The first new drug therapy developed exclusively for CF in 30 years was approved by the Food and Drug Administration (FDA) in 1993.  In clinical trials, this mucus-thinning drug called Pulmozyme®, reduced the number of respiratory infections and improved lung function.  In 1995, a four-year Foundation-supported study showed that the drug, ibuprofen, reduced the rate of lung inflmamation in children with CF - under controlled conditions, and in high doses.

In late 1997, the FDA approved the drug TOBI™ (tobramycin solution for inhalation).  In clinical trials, this reformulated version of the common antibiotic improved lung function in people with CF and reduced the number of hospital stays.  The benefits of TOBI™ are that it can be delivered in a more concentrated dose directly to the site of CF lung infections more efficiently, and that it is preservative-free.  The development of TOBI should lead to a long line of other aerosolized antibiotics for people with CF.

In addition, other treatment strategies to correct the protein product of the gene are currently being tested in clinical trials.  What makes these drugs unique is that researchers appear to be treating the causes of CF, not just the symptoms.

 

 

More Information

Cystic Fibrosis

Volunteer Information

Gene Therapy

615 N. Wolfe Street, Baltimore, MD 21205