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…at the Annual American Society of Hematology Meeting. “With a gene therapy like this, hopefully we can just people one injection and correct them forever.”
The technique, [Wang says], could potentially work on most patients with hemophilia B, the most common form of hemophilia. In theory, it would last years or even a lifetime—much longer than the arduous continual treatment most patients continually undergo.
The most exciting breakthrough here…[is that this strategy] may translate to other genetic conditions rooted in the liver, and potentially other organs, too.
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