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Cystic Fibrosis - Coggle Diagram
Cystic Fibrosis
Most common, fatal genetic disease in the United States
About 30,000 in the U.S. have it
What is it?
CF causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas.
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Possible treatment
Gene therapy
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Started in 1990 when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.
In 1993, the first experimental gene therapy treatment was given to a patient with CF . The scientists used a modified common cold virus to act as the delivery vehicle.
Studies have looked into other methods of gene delivery including fat capsules, nose drops, drizzling cells down a flexible tube to CFTR cells lining the airways of lungs, and aerosol delivery using nebulizers
Symptoms
very salty-tasting skin, a persistent cough, and excessive appetite but poor weight gain
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1 in 31 Americans is a symptom-less carrier of the defective CF gene, which they can pass on to their children.
To develop CF, a child must inherit a defective gene from both parents.
If both parents are carriers, there is a 25% chance that each of their children will have CF and a 50% chance that their child will be a carrier.