Researchers supported by the National Institutes of Health (NIH) have reported a potential new approach for treating certain forms of cystic fibrosis, using engineered transfer RNA delivered through lipid nanoparticles.

The research, published September 29, found that the treatment restored functional proteins in cell and animal models of cystic fibrosis. The approach is designed to address genetic mutations known as nonsense mutations, which can prevent cells from producing complete, functional proteins.

Cystic fibrosis is a genetic disorder caused by mutations in the CFTR gene. The disease affects nearly 40,000 people in the United States and can cause thick mucus to build up in the lungs and digestive system, contributing to breathing and digestive problems.

The researchers used lipid nanoparticles to deliver engineered tRNA into cells. In laboratory models, the strategy helped cells produce functional CFTR protein, suggesting a possible route for developing treatments for genetic forms of the disease.

The findings are preclinical, meaning the research has not established that the treatment is safe or effective for people. Further studies would be needed before the approach could become an approved human therapy.