How Do You Treat Cystic Fibrosis With Gene Therapy?
In 1989, researchers discovered CFTR, the gene that causes cystic fibrosis (CF). Gene therapy is still experimental, but scientists hope that this research will provide answers about treating the cause of cystic fibrosis rather than the symptoms. Gene therapy research has focused on lung cells because respiratory failure accounts for most cystic fibrosis deaths, but scientists hope to be able to use what they’ve learned to treat all affected genes. Learn about the Cystic Fibrosis Transmembrane Regulator (CFTR) gene that causes CF. To develop the disease, one defective gene must be inherited from each parent. Learn that in healthy people, the CFTR protein is found in the lining of major organs and controls the transport of chloride, salt and water across cell membranes. Salt dysregulation in CF patients results in a buildup of excess mucus that promotes bacterial growth, blocks glands and prohibits the movement of digestive enzymes. Study the treatment of cystic fibrosis with experiment
