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Test on ‘mini-organ’ gives people with cystic fibrosis a prognosis

Cystic fibrosis, also called cystic fibrosis (CF), is a serious genetic disease. People can also carry the gene abnormality without knowing it. If they also have a well-functioning gene in addition to the defective gene, they are not sick themselves. There are many variants, in which the clinical picture can differ.

Salty

What the genetic errors have in common is that the salt does not distribute properly in the body cells of people with cystic fibrosis. As a result, mucus in the body becomes tough. This can lead to serious health problems, such as pneumonia, diabetes and liver damage. The pancreas of patients also works less well.

Mini-organs

The research into the cells of CF patients has become easier because of the mini-organs. These are tiny spheres made from cells from the intestines of patients. Just like real intestines, the organs contain various parts, such as mini-pumps for the transport of substances.

Reliable size

“In cystic fibrosis, the pump function is disrupted,” explains researcher Danya Muilwijk. “What this study shows is that the size of these spheres, and thus the amount of fluid pumped in, is a reliable measure of the severity of the disease. Because the worse the disease, the less pump function, and therefore the smaller the spheres remain.”

Improved Medication

Half of the patients are currently not older than 50 years. Improved medication has made great progress in recent years and the results of the newest drugs are promising, according to doctors. The mini-organs can also play a role here. Existing medicines and medicines that are still under development can be tested with the mini-organs. “Without patients having to test all kinds of drugs themselves, the clinical effects of which are not always clear and which involve many hospital visits,” adds Professor Jeffrey Beekman. (ANP)


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