Research News from CHOP
Preclinical Study from Children’s Hospital of Philadelphia Highlights Innovative Approach to Replacing Brain Cells in the Treatment of Leukodystrophies
Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania announced the first ever direct approach to the depletion and replacement of microglia, the native immune cells of the central nervous system (CNS).
FDA Approves Gene Therapy for Epidermolysis Bullosa
ZEVASKYN™ is an ex vivo gene therapy approved for pediatric recessive dystrophic EB patients, where a genetically modified skin graft is applied to help wound healing.
Children’s Hospital of Philadelphia’s First FDA Approved Gene Therapy Patient for Severe Beta Thalassemia Celebrates Two Year Treatment Anniversary
Patient no longer needs regular blood transfusions for her inherited blood disorder, and her future has been transformed.
Children’s Hospital of Philadelphia Researchers Develop Specialized Growth Charts to Help Doctors Track Rare Pediatric Overgrowth Syndrome
Researchers from Children’s Hospital of Philadelphia (CHOP) collaborated with other health systems to develop specialized growth charts for children with Beckwith–Wiedemann Spectrum (BWSp), the most common epigenetic overgrowth and cancer predisposition disorder in children. The findings illustrate how newly developed growth charts are designed to better represent how children with the condition grow, which can improve personalized treatment.
Children’s Hospital of Philadelphia Researchers Find Gene Therapy Shows Long-Term Benefits for Hemophilia B Patients
Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity.
Children’s Hospital of Philadelphia Researchers Develop Cell Atlas to Uncover Significant Shifts in the Neuroblastoma Tumor Microenvironment
Researchers at Children’s Hospital of Philadelphia (CHOP) developed a longitudinal atlas of neuroblastoma, a common and potentially deadly childhood cancer, to gain deeper understanding into precise molecular mechanisms underlying why and how certain treatments eventually become ineffective.
Children’s Hospital of Philadelphia Researchers Shed Light on How Rare Pediatric Overgrowth Syndrome May Raise Cancer Risk
New research from Children’s Hospital of Philadelphia (CHOP) offers insight into how a rare genetic condition called Beckwith–Wiedemann syndrome (BWS) affects the liver and may raise cancer risk.
Children’s Hospital of Philadelphia-led Pilot Program Aims to Ease Burnout Among School Staff in Under-Resourced Schools
To reduce staff stress and burnout, researchers piloted a 12-session resilience program in two West Philly K-8 schools and evaluated its impact and adaptability.
Researchers at Children’s Hospital of Philadelphia Show Gene Therapy Can Improve Quality of Life for Companion Dogs with Hemophilia
In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.
Children’s Hospital of Philadelphia Researchers Discover that Acute Myeloid Leukemia Can Permanently Reprogram Stem Cells Through Inflammation
New research from Children’s Hospital of Philadelphia (CHOP) uncovered a previously unknown side effect of Acute Myeloid leukemia (AML), the second most common blood cancer in children, that leaves a long-lasting imprint on healthy blood-forming stem cells, influencing immune responses long after the disease has entered remission. The results were published recently in the journal Cancer Letters.