Researchers have been able to use CRISPR gene editing to fix a typically lethal liver condition in mice in utero, Stat News reports.
A Children's Hospital of Philadelphia-led team used base editing, a form of CRISPR-based genome editing, to target the PCSK9 gene, in which loss-of function mutations lead to low cholesterol levels and decreased coronary heart disease risk. As they report in Nature Medicine this week, making this change in fetal wild-type mice led to mouse pups with lower levels of the PCSK9 and of total cholesterol.
The researchers then targeted the HPD gene in a fetal mouse model of hereditary tyrosinemia type 1, which is lethal to neonates, to introduce a nonsense mutation. This treatment rescued the lethal phenotype in mice, the researchers report.
In all, the researchers say their proof-of-concept work shows that fetal gene editing could be a viable treatment. "A lot more animal work needs to be done before we can even think about applying this [fetal genome editing] clinically," CHOP's William Peranteau tells Stat News. "But I think fetal genome editing may be where fetal surgery [which is now routine] once was, and that one day we'll use it to treat diseases that cause significant morbidity and mortality."