Question

Explain how gene therapy might be used to treat cystic fibrosis

Answer 1

cystic fibrosis is caused by a mutation to the gene for the CFTR protein which is involved in chloride transport across membranes. Gene therapy would involve correcting the mutation in the genome of all cells or at least the cells involved in mucous secretion in the lungs so that all or at least the most significant symptoms of the disease (consolidation, repeat infection and severe bronchiectasis in the lung) can be avoided. The difficulty in this is getting the correct allele into the relevant cells, into the genome and stimulating expression appropriately, this cannot be done currently and is an area of intense research