KU Leuven achieves Belgian breakthrough in fight against cystic fibrosis
By overriding mutations in a defective gene, researchers at KU Leuven have managed to obtain a perfectly healthy gene. This development effectively cured cystic fibrosis in human airway cells, at least in the lab.
There are about 1,400 CF patients in Belgium. Cystic fibrosis, also called mucoviscidosis, is the most common congenital and life-threatening condition. As a result of impaired salt and water transport, tough mucus accumulates in the respiratory tract and a variety of organs. It is a degenerative disorder that includes a chronic cough, lung infections, diarrhoea, abdominal pain, intestinal blockages and more. Medication, aerosol and physical therapy are used to try to control this incurable disease. Sometimes a lung transplant may be required.
Prime editing technology, however, should change that within a few years. This fairly recent therapy allows the mutated gene to be cut away and replaced. It is a very precise and safe process, with no accidental collateral damage in the DNA. For its tests, the KU Leuven lab removed a piece of tissue from a CF patient. From this biopsy, it grew a so-called organoid, which is a microscopic organ that mimics both the structure and function of the real organ from which it was sourced.
The key question now is how to successfully take this genetic modification from the petri dish to the body. That will require additional research and time, but things are looking hopeful for CF patients.