A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly into human cells and can repair the chloride channel most commonly affected in cystic fibrosis. The innovative therapeutic approach was developed in collaboration between teams from Charité—Universitätsmedizin Berlin and the Leibniz-Forschungsinstitut für Molekulare Pharmakologie (FMP). The results have now been published in the journal Nature Chemical Biology.
The clinical picture of cystic fibrosis—also known as CF—is caused by genetic defects in the so-called CFTR channel. This channel regulates water and salt transport in the lung mucosa and ensures the production of sufficiently fluid mucus. In about 90% of cystic fibrosis patients, a mutation known as F508del is present in the CFTR channel, meaning that a single amino acid is missing at position 508 in its protein chain. This change causes CFTR to fold incorrectly and break down prematurely inside the cell, rather than functioning as a channel in the cell membrane of the airways.
As a result, patients have thick mucus in their lungs, and pathogens can no longer be effectively cleared. The consequence is chronic infection and inflammation of the airways, leading to a progressive loss of lung function—in the worst-case scenario, this necessitates a lung transplant.








