There are several problems with this approach. Firstly, release factors that recognize nonsense mutations are likely to prevent, most if not all, CFTR protein synthesis. Secondly, it is not known whether this CFTR protein, if produced, will be effective in restoring normal ion transport function in vivo. Other recent approaches in treating CF include ion transport and anti-inflammatory therapies. However, since mutant CFTR protein is usually retained within the endoplasmic reticulum and is not delivered to the cell surface membrane, this pharmacological treatment is unlikely to be effective.
Lung transplantation is an option for people with end-stage respiratory failure.
This leads to dehydration of mucus outside the cells. The obstruction of the airways caused by thick, dehydrated mucus supports repeated microbial infection, especially with Pseudomonas species, which often leads to chronic inflammation of the airways and death. The association between excessive mucus production and the CF mutation remains unclear. There is, however, an hypothesis that increased sodium absorption and lack of cAMP-mediated chloride transport resulting from the CFTR defect, causes reduced mucociliary clearance . If this hypothesis is correct, the rectification of the defective ion transport function in lung epithelia with a normal CFTR gene would lead to the ultimate cure for cystic fibrosis.