Tag Archive: gene editing

Apr 19 2019

In the News: PAS 2019, NFL Helmet Safety, American Society of Gene and Cell Therapy, Bypass Prophylaxis Hemophilia, Lung Disease In Utero

Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows. This week, read about what to expect at next week’s Pediatric Academic Societies Meeting, discover how a CHOP engineer is helping to make the NFL safer, and learn about new results from our Center for Fetal Research about treating lung diseases in utero.

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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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Dec 15 2017

Brain Plasticity, Hemophilia B Gene Therapy, Stem Cells and SCD, Medical Cannabis for Autism, PA Rare Disease Advisory Council

The year 2017 might be coming to a close, but research continues to ramp up at Children’s Hospital of Philadelphia, with exciting developments in the fields of brain science, hemophilia, gene therapy, and more. In this week’s roundup of headlines, we take a look at remarkable reports from CHOP and Penn Medicine about the brain’s ability to reorganize itself after limb amputation, the first U.S. effort to observe the use of medical cannabis for children with autism, and exciting innovations to improve sickle cell disease treatment presented at the 59th American Society of Hematology Annual Meeting and Exposition. Read on to discover more about these brilliant breakthroughs. 

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