Question

In gene therapy, DNAase is used to treat

• A. Cystic fibrosis
• B. Haemophilia
• C. Pituitary dwarfism
• D. Insulin dependent diabetes

Answer 1

The correct option is A Cystic fibrosis

DNase is an experimental drug used to treat cystic fibrosis. It is an autosomal recessive disease that affects lung, liver, pancreas, kidneys and intestine. It is caused due to the mutations in both copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR). It was found that the mucus of the patient is full of DNA, which spills out of white blood cells. DNase cuts the DNA in the mucus. It is delivered in the form of an aerosol in the patient and it improves lung function by breaking up the thick mucus in the lungs. The patient is then able to clear the lungs by coughing.

So, the correct answer is option A.