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

CF affects approximately 30,000 children and young adults.  It occurs in approximately one of every 3,200 live Caucasian births (in one of every 3,900 live births of all Americans.)  There are about 1,000 new cases of CF diagnosed each year.  Most individuals are diagnosed by the age of three, however, nearly eight percent of all newly diagnosed cases are 18 or older.  According to the CF Foundation's National Patient Registry, one half of all individuals with CF live to the age of 31, one half do not.

One in 31 Americans (one in 28 Caucasians) - more than 10 million people - is an unknowing, symptomless carrier of the defective gene.

An individual must inherit a defective copy of the CF gene - one from each parent - to have cystic fibrosis.  Each time two carriers conceive a child, there is a 25 percent chance that the child will have CF; a 50 percent chance that the child will be a carrier, and a 25 percent chance that the child will be a non-carrier.

CF has a variety of symptoms.  The most common are: very salty-tasting skin; persistent coughing, wheezing or pneumonia; excessive appetite but poor weight gain; and bulky stools.

The basic defect in CF cells is the faulty transport of sodium and chloride (salt) within epithelial cells - which line organs such as the lungs and pancreas - to their outer surfaces.

CF causes the body to produce an abnormally thick, sticky mucus.  This abnormal mucus clogs the lungs and leads to fatal infections.  The thick CF mucus also obstructs the pancreas, preventing enzymes from reaching the intestines to digest food.

The treatment of CF depends upon the stage of the disease  and which organs are involved.  One means of treatment, postural drainage (also called chest physical therapy [CPT]), requires vigorous percussion (by using cupped hands) on the back and chest to dislodge the thick mucus from the lungs.  Antibiotics are also used to treat lung infections and are administered intravenously, via pills, and/or medicated vapors which are inhaled to open up clogged airways.  When CF affects the digestive system, the body does not absorb enough nutrients.  Therefore, people with CF may need to eat an enriched diet and take both replacement vitamins and enzymes.

 

 

More Information

Volunteer Information

Treatments

Gene Therapy

615 N. Wolfe Street, Baltimore, MD 21205