Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

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.