A win for prenatal CRISPR

In an early-stage study on mice, scientists managed to cure an inherited disease by using CRISPR genome editing on fetuses, a promising sign that the rapidly proliferating technology may offer hope for devastating inborn disorders.

As STAT’s Sharon Begley reports, researchers at the University of Pennsylvania and the Children’s Hospital of Philadelphia managed to cure a disease called hereditary tyrosinemia type 1 in mice by disabling an errant gene, an early victory that could pave the way to curing genetic disorders before birth.

“A lot more animal work needs to be done before we can even think about applying this clinically,” said Dr. William Peranteau, who co-led the study. “But I think fetal genome editing may be where fetal surgery once was, and that one day we’ll use it to treat diseases that cause significant morbidity and mortality.”

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