Using CRISPR gene editing, a team from Penn Medicine and Children’s Hospital of Philadelphia have thwarted a lethal lung disease, in which a harmful mutation causes death within hours after birth. This proof-of-concept study, published in Science Translational Medicine, showed that in utero editing could be a promising new approach for treating lung diseases before birth.
“We wanted to know if this could work at all,” says study co-leader Edward E. Morrisey, a professor of cardiovascular medicine and cell and developmental biology in the Perelman School of Medicine, and scientific director of Penn’s Institute for Regenerative Medicine. “The trick was how to direct the gene-editing machinery to target cells that line the airways of the lungs.”
The lung conditions the team is hoping to solve—congenital diseases such as cystic fibrosis, surfactant protein deficiency, and alpha-1 antitrypsin—are characterized by respiratory failure at birth or chronic lung disease with few options for therapies. About 22 percent of all pediatric hospital admissions are attributed to respiratory disorders, and congenital causes of respiratory diseases are often lethal, despite advances in care and a deeper understanding of their molecular causes. Because the lung is a barrier organ in direct contact with the outside environment, targeted delivery to correct defective genes is an attractive therapy.
Read more at Penn Medicine News.