Gene therapy delivers lasting immune protection in children with rare disorder
Peer-Reviewed Publication
Updates every hour. Last Updated: 29-Dec-2025 20:11 ET (30-Dec-2025 01:11 GMT/UTC)
A new collaborative study from the Wyss Institute leveraged a “breathing” human lung alveolus chip model of influenza A infection developed in the group of Donald Ingber, M.D., Ph.D., drug delivery platforms advanced by Natalie Artzi, Ph.D. and her group, as well as state-of-the-art CRISPR technology. The team designed CRISPR machinery targeting a strongly conserved sequence in IAV’s genome, packaging it up in tiny nanoparticles with affinity to lung epithelial cells, and delivering the loaded particles to lung epithelial cells lining a microfluidic channel in the Lung Chip that were infected with a pandemic IAV. They demonstrated that this system better mimics human IAV infection than other preclinical models and enables assessing the efficacy and safety of CRISPR RNA therapies in a more clinically relevant way than earlier approaches.