Scientists have used a gene editing tool to thwart a lethal lung disease in animals in which a harmful mutation causes death within hours after birth.
The proof-of-concept study, published in the journal Science Translational Medicine, showed that in utero editing could be a promising new approach for treating lung diseases before birth.
“The developing foetus has many innate properties that make it an attractive recipient for therapeutic gene editing,” said William H Peranteau, an investigator at Children’s Hospital of Philadelphia (CHOP) in the US.
“Furthermore, the ability to cure or mitigate a disease via gene editing in mid- to late gestation before birth and the onset of irreversible pathology is very exciting. This is particularly true for diseases that affect the lungs, whose function becomes dramatically more important at the time of birth,” Peranteau said in a statement.
The lung conditions the team is hoping to solve—congenital diseases such as surfactant protein deficiency, cystic fibrosis, and alpha-1 antitrypsin—are characterised by respiratory failure at birth or chronic lung disease with few options for therapies.
About 22 per cent of all pediatric hospital admissions are because of respiratory disorders, and congenital causes of respiratory diseases are often lethal, despite advances in care and a deeper understanding of their molecular causes.
