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.
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