Nanobody Repairs Misfolded Protein in Cystic Fibrosis Cells for First Time

Researchers have for the first time developed a nanobody that penetrates human cells and repairs the misfolded CFTR protein responsible for most cystic fibrosis cases, boosting chloride-channel function in laboratory tests. The small antibody fragment can reach intracellular targets that conventional antibody drugs cannot, opening a new class of therapies for genetic diseases rooted in protein-folding defects. The breakthrough could complement existing cystic fibrosis modulator drugs and potentially reach patients whose specific CFTR mutations don't respond to current treatments, a population that has long been left behind by first-generation precision therapies.