Cystic Fibrosis Gene Therapy: A Treatment Worth Considering?

Abstract

In recent years, gene therapy has emerged as a novel form of treatment for many genetic illnesses, one of the most notable being cystic fibrosis (CF). Gene therapy can be used to deliver a correct, non-mutated copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene into the body via a virus cell to replace the mutated copy of the CFTR gene in CF patients, which causes the illness. If done correctly, gene therapy can offer a non-invasive, versatile treatment that can cure cystic fibrosis at the source (or in other words, at the mutation). However, the viral vectors used to transport the CFTR gene can cause fatal inflammation and infection, along with the vectors attacking the wrong tissue. These two deadly side effects in conjunction with the hundreds of thousands dollar price tag make CF gene therapy an ineffective form of treatment.