Approximately 70,000 people live with Cystic Fibrosis, a hereditary respiratory disorder caused by faulty genes. That number is growing; an estimated 1,000 new cases of CF are documented each year.
Fortunately, scientists around the world are working on a new treatment called gene therapy. In this therapy, “copies of a normal gene are inserted into the DNA of cells to counter the effects of the faulty gene.”
16 year old Mary Bondonno participated in one of the recent clinical trials. Due to cystic fibrosis, Mary has to undergo an extensive hour long treatment every morning just to manage her symptoms. The treatment includes breathing exercises, nebulizers, inhalers, and physiotherapy. But once Mary started receiving gene therapy, she found it made a huge difference, “I had a lot more energy, I didn’t need my wheelchair, my grades were going up, and I was getting a lot more done,” she said.
Unfortunately, once the trail stopped, so did her gene therapy. “It was the best year of my life. So I want that back.”
Currently, scientists are looking for ways to amplify and enhance gene therapy, as well as trying to make the treatment more available. One new strategy for enhancement is called cationic liposome. In this strategy, DNA is delivered to the faulty gene via a fatty container which delivers DNA directly to a cell. Initial results “show promise.”
Hopefully, we will soon live in a world where cystic fibrosis is more manageable thanks to gene therapy.