Tag Archive: SPK-9001

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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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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Aug 24 2018

In the News: For the Love of Grace Foundation, Hemophilia Research in the NYT, Microbiome Symposium, Baby Monitors, 2018 Parkway Run

Microbes, monitors, and miraculous medicine converge in this week’s roundup of research-related headlines at Children’s Hospital of Philadelphia. Keep reading to learn more about new concerns for commercially available baby monitors reported by our researchers, one family’s inspirational story of resilience and recovery after their baby daughter battled a brain tumor, and exciting upcoming events in the CHOP community, including the fifth annual symposium for microbiome research.

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