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Editing in the Womb

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."

The Scan

Foxtail Millet Pangenome, Graph-Based Reference Genome

Researchers in Nature Genetics described their generation of a foxtail millet pangenome, which they say can help in crop trait improvement.

Protein Length Distribution Consistent Across Species

An analysis in Genome Biology compares the lengths of proteins across more than 2,300 species, finding similar length distributions.

Novel Genetic Loci Linked to Insulin Resistance in New Study

A team reports in Nature Genetics that it used glucose challenge test data to home in on candidate genes involved in GLUT4 expression or trafficking.

RNA Editing in Octopuses Seems to Help Acclimation to Shifts in Water Temperature

A paper in Cell reports that octopuses use RNA editing to help them adjust to different water temperatures.