Cystic fibrosis is an inherited disease of the secretory glands that are responsible for producing mucus and sweat. Parents may not even have the disease themselves, but will still pass faulty genes to their children. Now a new research, involving an international team of scientists, identified molecules that could help prevent cystic fibrosis and other diseases caused by faulty ion channels.
Cystic fibrosis is characterized by impaired transportation of chloride ions. It is caused by faulty ion channels that are supposed to create minute openings in the cell membrane for the movement of specific ions. There are ways to treat this condition – scientists have created synthetic transporters to move chloride ions through lipid-bilayer membranes.
However, these anion transporters are far from perfect, as they also transport protons or hydroxide ions and can potentially disrupt pH homeostasis and even have some toxic effects. Now a new research found a new kind of transporters that are capable of selecting chloride ions with high accuracy. This is the first research of its kind to demonstrate that undesired proton/hydroxide transportation is an overlooked side-effect, which should be addressed.
Scientists managed to synthesise two new molecules, which are extremely selective when carrying chloride ions. One of these molecules is already tested to be able to transport chloride ions without having big effect on lysosomal pH.
Xin Wu, lead author of the study, said: “These new findings represent a paradigm shift for transporter design and provide important clues on how to develop anion transporters for different biomedical applications. We showed that different classes of anion transporters can have different behaviour in regulating ion gradients, membrane potential and pH gradients in cells”.
Development of replacement molecules for faulty chloride channels without disrupting pH homeostasis is a big step towards new therapies to treat cystic fibrosis and other diseases caused by poorly functioning ion channels. However, before this new generation of anion transporters can be used more research has to be done, which may take some time.