Cystic Fibrosis and Gene Therapy
Cystic fibrosis is a genetic disorder that affects the gene CFTR. CFTR is responsible for coding a protein that controls the channels that water and chloride flow from. The channels will either be non-existent or they will function very poorly. The disease will cause difficulty breathing due to a build up of sticky mucous. It causes poor weight gain, recurring chest and lung infections, and a persistent cough and usually begins in childhood. Cystic fibrosis does not have any treatment, although there are treatments to help alleviate symptoms. Studies that have been conducted before have been unsuccessful in trying to see if gene therapy could work as treatment.
Gene therapy is a method in which a gene that is causing a problem is replaced by copy that is healthy. Professor Zeger Debyser, a professor from KU Leuven, explained a recent study that was conducted on mice. This study used recombinant adeno-associated virus (rAAV), which will insert a healthy copy of the CFTR gene. The study showed an improvement in mice as well as in cultures of gut cells from patients with cystic fibrosis. The flow of chloride and water ions was fully restored, however there is more research that needs to be done. Researchers now have to observe how long the treatment works, and determine whether or not more than one treatment will be necessary. I think that this is big breakthrough in the study of treatments for cystic fibrosis, but there is a lot more research to be done. I believe that gene therapy has proven to be successful in some people, but not in others. I look forward to the evolvement of gene therapy and the treatments that it is going to develop.