Tag Archive: Spark Therapeutics

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.

Read the rest of this entry >>

Dec 29 2017

Gene Therapy Makes Headlines, Mitochondrial Medicine, Brain Injury Collaboration, Implicit Racial Bias Blog

After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!

Read the rest of this entry >>

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. 

Read the rest of this entry >>

Dec 16 2016

CHOP Research In the News: Global Trial of CTL019, Gene Therapy for Hemophilia, Child Abuse Within Military, New Radiologist-in-Chief, ADHD in Preschool

CHOP Research In the NewsYour holiday season has been hectic, no doubt. Catch up with an early gift from us: Our biweekly roundup of research news from Children’s Hospital of Philadelphia comes with all the trimmings!

Read the rest of this entry >>