At Children’s Hospital of Philadelphia, we know innovation sometimes requires a second look at seemingly harmless practices and a willingness to break out of the status quo. In this edition of In the News, learn how an unnecessary emergency room visit prompted Christopher Bonafide, MD, to examine the use of physiological monitors for healthy infants, and read about a bold move toward future innovation with the grand opening of our new Clinical Manufacturing Facility for precision medical tools. Additionally, the Center for Child Injury and Prevention Studies’ Annual Report highlights important safety work with real-world implications, a new Penn-CHOP collaboration aims to investigate nutritional interventions to treat disease, and a CHOP patient gets the surprise of a lifetime in the name of autism awareness.
The Human Genome Project’s successful completion 15 years ago gave us a new genomic lens to read our 20,000 or so protein-coding genes. Since then, a surge in next-generation sequencing technologies is generating new insights daily that sharpen our view of how the human genome works.
Yi Xing, PhD, was on the cusp of this revolution in medicine as he finished his PhD training in molecular biology and bioinformatics at University of California Los Angeles (UCLA). His research career began to rise during the incredible takeoff of big data science, and he became a prominent scientist in this cutting-edge field. The immense challenges of synthesizing diverse data sets from many sources come with vast opportunities to change pediatric medicine, which is why Dr. Xing is eager to assume his new role as the inaugural director of the Center for Computational and Genomic Medicine at Children’s Hospital of Philadelphia.
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.
The scientific wonder of stem cell research and its implications for medicine have come a long way in the last decade: At Children’s Hospital of Philadelphia Research Institute, our investigators’ innovative use of stem cell science to approach complex pediatric conditions continues to inspire for their potential to improve outcomes in children’s health. In our latest news roundup, learn about novel stem cell research from our Cancer Center and Division of Urology that aims to preserve the future fertility of boys who undergo childhood cancer treatment. Discover a new project co-led by a CHOP neurology researcher that takes a stem cell approach to restore vision cells in blind dogs.
PolicyLab hosted a 10th anniversary celebration Oct. 22 that its attendees won’t forget. After more than a decade of working to inform policies impacting children’s health, the Center of Emphasis at Children’s Hospital of Philadelphia gathered regional and national leaders alongside clinicians, academics, and community collaborators at the National Museum of American Jewish History in Philadelphia to discuss the future of children’s health policy and practice.
With the release of over 200 genomic tumor models spanning 25 different types of childhood cancer, researchers may now have the ability to skip lengthy preclinical work in their development of novel treatments. With funding from Alex’s Lemonade Stand Foundation (ALSF), the Pediatric Preclinical Testing Consortium (PPTC) announced their data sets will now be made available to any qualified academic petitioner — a move that John Maris, MD, oncologist at Children’s Hospital of Philadelphia Cancer Center and principal investigator of the PPTC’s CHOP site, believes is the first of its kind.
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.
Four scientists from Children’s Hospital of Philadelphia’s Cancer Center received grants from the St. Baldrick’s Foundation to conduct research projects to improve outcomes for children with cancer.
The top private funder of childhood cancer research grants in the U.S., St. Baldrick’s awarded researchers and institutions around the nation a total of $19.1 million this year in what the charitable organization called “its biggest grant cycle of 2018.” In all, St. Baldrick’s has funded more than $253 million in childhood cancer research grants since 2005.
With an enterprising career spanning 50 years in basic and clinical immunology and more than 500 publications, Steven D. Douglas, MD, had the honor of presenting the 25th Herman and Gertrude Silver Lecture, in which he reviewed major milestones in the field of pediatric HIV/AIDS and shared his optimism that paradigm shifts and new discoveries are ahead.