Tag Archive: William H. Peranteau

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.

Read the rest of this entry >>

Oct 19 2018

In the News: Sepsis in Scientific American, Updating Genetic Tests, Prenatal Gene Editing, Nature Magazine, Type 1.5 Diabetes

Our breakthrough research at Children’s Hospital of Philadelphia Research Institute made headlines in both popular science media outlets and prominent scientific journals alike, from advances in detecting sepsis, to pioneering work in prenatal gene editing, to the future of genomic medicine. Read on to learn more about how our scientists stay at the cutting-edge of their fields by approaching pediatric medicine inventively, whether it’s by developing the most effective alerts in emergency medicine, modernizing genetic testing models to keep up with an accelerating pace of discovery, and beyond. 

Read the rest of this entry >>