EVERYDAY SCIENCE

Detailed explanation-1: -Integrating Gene Therapy This kind of gene therapy is like binding a new page into an existing book. An advantage of an integrating gene therapy is that it is permanent for the life of the cell. This means that a person with CF might have to receive the gene therapy only once or a few times in their life.

Detailed explanation-2: -Over 1, 700 different mutations have been identified in the CFTR gene that can cause cystic fibrosis, and gene therapy may be the most effective way to combat all of them at once. Nonetheless, gene therapies for treating cystic fibrosis are still in their early stages of development.

Detailed explanation-3: -People with cystic fibrosis have a mutation in both copies of the cystic fibrosis membrane conductance regulator (CFTR) gene. To correct the CFTR mutations in someone’s DNA, the tools needed for gene editing need to get inside the person’s cells, which is very challenging.

Detailed explanation-4: -How does AAV work? Simply put, AAV is transformed from a naturally occurring virus into a delivery mechanism for gene therapy. The viral DNA is replaced with new DNA, and it becomes a precisely coded vector and is no longer considered a virus, as most of the viral components have been replaced.