Embarrassing Illnesses

Since the faulty gene that causes cystic fibrosis was identified, scientists have been carrying out research to try to correct the genetic defect. This is known as gene therapy, which replaces faulty genes with normal ones.

How gene therapy works

A complete set of genes are present in every cell of your body (except red blood cells), and these genes contain all the information needed to build a human being. It is your genes that determine factors like the colour of your eyes, and whether or not you can roll your tongue.

Each human cell contains 46 chromosomes, which are made up from 23 chromosomes from your mother, and 23 from your father. There are approximately 30,000 genes on each chromosome, and the faulty gene that causes cystic fibrosis is found on chromosome 7. For a child to be born with cystic fibrosis, they must inherit a copy of the faulty gene from both parents.

Gene therapy for cystic fibrosis involves adding a healthy copy of the faulty gene into the relevant cells in the body, such as the ones in the lungs and digestive system. This can be done using either viruses, which have been developed to enter the cells efficiently, or liposomes, which are fatty substances that naturally stick to the surface of cells and encourage genes to enter them.

So far, the benefits of gene therapy have only been made to last for a few days. However, with the knowledge that gene therapy works, scientists can continue to research how to make the effects permanent. The two main challenges for them are getting the healthy genes into the cells in a more efficient way, and making the benefits last as long as possible.