“Hemophilia is a devastating genetic condition—without the ability to form blood clots, those who have it risk bleeding to death from even the slightest cut.
But scientists at the University of Pennsylvania have their sights set on a potential cure. Working in mice, they used the gene editing technique CRISPR to repair defective genes in a clotting protein called factor IX, the source of the mutations that cause hemophilia B. And it worked.
“Basically we cured the mice,” Dr. Lili Wang, the lead author on a forthcoming paper presented Monday at the Annual American Society of Hematology Meeting, told me. “With a gene therapy like this, hopefully we can just people one injection and correct them forever.”