Researchers are reporting development of a new delivery tool for gene-editing that targets a broad range of lung diseases including cystic fibrosis, COPD, and asthma.
Scientists are excited about the possibility of using gene editing to treat diseases by repairing or modifying disease-causing mutations. But one of the hurdles remains how to deliver these tools to the right cells in patients safely and efficiently.
In the new study, researchers used simple peptides to delivery gene-editing tools into notoriously hard-to-access lung and airway cells with the goal of treating lung diseases. They showed that these cell-penetrating “shuttle” peptides can carry gene-editing proteins into airway cells without causing harm to the cells or the animal model tested and do so at levels that might be useful clinically.
Their study, funded in part by NHLBI, appeared in Nature Communications.