How would you use gene therapy to treat CF What problems mig

How would you use gene therapy to treat CF? What problems might the plasma membrane cause for successful gene therapy? (5 points)

Solution

Cystic fibrosis is a heredity disorder affecting the exocrine glands. It cause the production of abnormally thick mucus, leading to the blockage of the pancreatic ducts, intestines and bronchi and often resulting in respiratory infection.

Cystic fibrosis is an autosomal recessive, life limiting disease resulting from mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In normal cells, the CFTR channel protein helps on the transport of chloride ions but in the mutated CFTR (in cystic fibrosis), the CFTR fails to transport chloride ions across the membrane. As a result very few chloride ions move into the mucous layer and too many sodium ions move into the cell. The movement of sodium ions into the cells causes water to flow in reverse direction and flow back into the cell, thus drying the mucus layer. The mucus layer becomes thick and dry. This causes individuals with cystic fibrosis. The cystic fibrosis can be treated by gene therapy.

Gene therapy is the introduction of the normal genes into the cells in place of missing or defective one in order to correct the genetic disorder.Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator(CFTR) DNA to the epithelial cells in the airways. Also, it is feasible to deliver the gene to the affected area i.e. lungs.