Cystic fibrosis is a hereditary disease caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR gene encodes an ion channel protein that is involved in the transport of salts across cell membranes. Any of dozens of different mutations of the gene can lead to the development of cystic fibrosis.
The primary symptom of cystic fibrosis is a build-up of mucus in the lungs and other organs, leading to frequent infections and other complications. The lifespan of a person with cystic fibrosis is significantly shortened by the disease.
Gene Therapy Studies
Gene therapy may offer a potential avenue for curing cystic fibrosis through the repair of the defective CFTR gene. The basic idea behind gene therapy is to deliver a functional copy of the gene to cells and program them to begin making the functional copies. There are