What is gene therapy used for in relation to cystic fibrosis?

Gene therapy for cystic fibrosis aims specifically at correcting or addressing the underlying genetic issues that lead to the disease. In this context, introducing new genes into the lungs via an inhaler is a significant method in research and treatment strategies. By inhaling the therapeutic genes, healthy copies of the CFTR gene (which is defective in cystic fibrosis patients) can be delivered directly into the cells lining the lungs.

This approach seeks to produce functional CFTR proteins, which can help restore the balance of salt and water on the cell surface, ultimately improving lung function and reducing the severity of symptoms associated with cystic fibrosis. This targeted delivery mechanism is part of ongoing efforts to develop effective gene therapy treatments for cystic fibrosis that can provide a more practical and immediate approach to managing the condition compared to other methods that might require more invasive procedures or broader systemic treatment.