Oct 8 2018

The Science Behind Gene Therapy: Creating Breakthroughs One Gene at a Time

For more than 20 years, researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania have been at the forefront of taking a system perfected by nature — a virus — and transforming it into breakthrough gene therapies for rare single-gene diseases. CHOP was the first pediatric research institution to develop chimeric antigen receptor T cell (CAR-T) therapy for acute lymphoblastic leukemia. In this approach, viral vectors are used to modify a patient’s own T cells, training them to track down and eliminate the circulating cancer cells.

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Oct 5 2018

In the News: Silver Award, Supporting Outcomes Research, Hope on Wheels, Home Visiting Programs

As we move full-speed ahead into fall, the new season brings with it a handful of headlines from our researchers at Children’s Hospital of Philadelphia. This week, we share the latest news about investigators who have been recognized for their research legacies and for their current cutting-edge work, from discoveries in the basic and clinical science of HIV/AIDS, to the development of learning health system-based training in outcomes research, to the design of innovative approaches to childhood cancer.

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Oct 3 2018

Bone and Mineral Research Award Celebrates Career of Michael A. Levine, MD


For Michael A. Levine, MD, a pediatric endocrinologist, scientist, and fixture in the bone and mineral research community for nearly four decades, few things are a greater source of pride than to be honored by one’s own peers. The medical director of the Center for Bone Health at Children’s Hospital of Philadelphia and chief emeritus of the hospital’s Division of Endocrinology and Diabetes, was recognized Sept. 29 by the American Society of Bone and Mineral Research (ASBMR) — an organization he describes fondly as his “home society” — with the 2018 Frederic C. Bartter Award.

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Oct 1 2018

What are Best Practices for Pediatric Nasogastric Tube Placement and Verification?

The findings:

Researchers have published best practice recommendations for nasogastric (NG) tube location placement and verification in pediatric patients. Endorsed by the American Society for Parental and Enteral Nutrition (ASPEN), the recommendations include: provide education; use appropriate NGT placement and securing methods; measure gastric pH; consider a radiograph for any patient in whom there is a concern for correct NGT placement; and improve interpretation and communication about the radiograph.

Why it matters:

Upto this point, no standard for the NG tube insertion procedure existed in the United States. If performed improperly, the common practice has potentially serious or fatal complications, including tubes being placed in the child’s lung or snaking into the brain. In addition, the tube can be inserted into the small intestine rather than the intended target of the stomach.

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Sep 28 2018

New Website Opens Digital Door for Improving Parent, Teen Communication


Editor’s Note: It’s an exciting time for the Center for Parent and Teen Communication (CPTC) at Children’s Hospital of Philadelphia. The Center launched a new website, www.parentandteen.com, offering timely, easy to understand articles, videos, podcasts, slideshows, quizzes, and more for both parents and teens. For parents and caregivers, topics include: developmental forces that shape adolescent behavior, strategies for improving parent-teen communication, avenues for supporting character strengths that lead to a successful life, and best approaches for disciplining and monitoring. We took the opportunity to ask Kenneth Ginsburg, MD, MSEd, co-founder and director of programs for the CPTC, to tell us more about the Center and its research efforts.

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Sep 26 2018

Looking Back on the Partners for Child Passenger Safety Program

In 1997, researchers at Children’s Hospital of Philadelphia partnered with State Farm Insurance, one of the largest automobile insurance companies in the U.S., to create the nation’s first large-scale, child-focused crash surveillance system. Over the next 10 years, the Partners for Child Passenger Safety Program (PCPS) studied more than 875,000 children involved in 600,000 car crashes, conducted 33,000 in-depth interviews, and analyzed over 800 on-site investigations. By its close in 2007, the world’s largest study of children in crashes had produced a robust set of recommendations based on hard data and backed by scientific expertise that would shape and advance legislation, vehicle design, and public health education for years to come. Since PCPS began, the U.S.

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Sep 24 2018

Global Health Researchers Finding Ways to Improve Children’s Cancer Survival Rates

Every child with cancer deserves the greatest opportunity to be cured, no matter where in the world they live. This overarching sense of purpose took Julianne Burns, MD, a third-year pediatric Infectious Diseases fellow, from Children’s Hospital of Philadelphia to the Hospital Infantil Dr. Robert Reid Cabral (HIRRC) in the Dominican Republic for four weeks this summer to conduct much needed infectious disease research.

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Sep 21 2018

In the News: Brain Cancer Gene Therapy, 2019 Eagles Autism Challenge, Mitochondrial DNA in Cardiomyopathy, Kids First DRC, Device Symposium

Fall weather and football season have returned to us here at Children’s Hospital of Philadelphia Research Institute, bringing with them a fresh batch of research headlines. In this edition of our biweekly news roundup, catch up on the latest announcements for the second annual Eagles Autism Challenge, learn about new insights into the role mitochondrial DNA plays in heart disease progression, and stay updated on how CHOP helps to drive medical innovation and entrepreneurship forward in the Philadelphia community and beyond.

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Sep 20 2018

Pioneering Novel Therapies for Hemophilia

Editor’s Note: Jacqueline Hunter, PhD, a postdoctoral fellow in the Wolfe Laboratory, wrote this article as part of the Advanced Career Exploration (ACE) Fellowship at Children’s Hospital of Philadelphia Research Institute. The ACE program gives fellows the opportunity to pursue projects beyond their main research focus. We’re especially excited to share Dr. Hunter’s work during National Postdoc Appreciation Week.

Normally, the body responds to a bleeding event by forming a clot, which is a complicated process involving multiple proteins in an elegantly orchestrated cascade. When specific proteins of this cascade are absent, one of several debilitating disorders can occur that result in recurrent spontaneous bleeding into the joints and muscles. Researchers at Children’s Hospital of Philadelphia are using clever maneuvering to figure out new therapeutic options for patients with hemophilia A and B.

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Sep 18 2018

Studying Racial and Ethnic Disparities in Leukemia: Q&A with Richard Aplenc, MD

Acute myeloid leukemia (AML), the second most common leukemia in children, affects different populations of pediatric patients in different ways. With the support of a new Epidemiology Grant from Alex’s Lemonade Stand Foundation, Richard Aplenc, MD, PhD, assistant vice president and chief clinical research officer at Children’s Hospital of Philadelphia, is leading a powerful research project based on the automated extraction of data to learn more about racial and ethnic disparities observed in African American children with AML.

“We’ve known for a long time that African American children do worse with treatment for acute myeloid leukemia than non-African American children,” Dr. Aplenc said. “But we’ve never really understood why that is, and there are a lot of different possibilities.”

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