Tag Archive: Kiran Musunuru

Nov 9 2018

How Can Gene Editing Breakthroughs Help Prevent Congenital Disease Before Birth?

The findings:

In a first-of-its kind study, scientists performed prenatal gene editing in animals to prevent a lethal metabolic disorder and effectively open the door for similarly treating congenital diseases in humans before birth. Using gene editing technology, the team successfully targeted a gene that regulates cholesterol levels to lower cholesterol and, additionally, turned off the effects of a mutation that causes a lethal liver disease called hereditary tyrosinemia type 1 (HT1) in mice.

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