Alapati, D. et al. Sci Transl Med 11, eaav8375 (2019)

CRISPR-based gene editing therapies are making their way towards the clinic. There’s postnatal potential for treating a number of monogenic diseases, and new research in mice suggests the gene editing tool could work prenatally too.

Working with two mouse models of monogenic lung diseases that are lethal to pups within a few hours of birth, researchers have demonstrated that CRISPR-Cas9 vectors can correct mutations in developing mouse lungs in utero. Delivery was achieved via injection into the dam’s amniotic fluid, which is inhaled by the fetus and brings the tool to the developing lung tissue. Lung function and survival improved. Much remains to be refined, but the proof-of-concept study suggests that prenatal gene editing is feasible, an advance necessary to treat congenital diseases that are fatal to newborns.