A new study, led by researchers CHOP, identified tiny pieces of messenger RNA that are missing in pediatric high-grade glioma tumors but not in normal brain tissues. Preclinical research indicates that these missing RNA fragments can make difficult-to-treat tumors more responsive to immunotherapy. The findings were recently published in the journal Cell Reports.

A new study by the Center for Violence Prevention (CVP) at Children’s Hospital of Philadelphia, in collaboration with principal investigator Jungwon Min, PhD, MS from the University of Pennsylvania School of Nursing.

Researchers at Children’s Hospital of Philadelphia (CHOP), along with several academic partners, announced the primary results of the Pediatric KIDney Stone (PKIDS) trial, the largest comparative effectiveness study of surgical interventions for children and adolescents with kidney stones.

Researchers at Children’s Hospital of Philadelphia revealed insights into optimizing chimeric antigen receptor T cell (CAR-T) therapy for solid pediatric tumors, offering a more complete understanding of how tumor biology and immunological barriers affect efficiency and safety.

The Cardiac Center at the Children’s Hospital of Philadelphia (CHOP) is leading pioneering research with grants aimed at improving outcomes for children with severe cardiac conditions. These efforts represent a comprehensive approach to advancing cardiac care from prenatal stages through childhood, potentially transforming treatment paradigms.

Researchers at Children’s Hospital of Philadelphia (CHOP) are shedding light on how two genetic factors – an extra copy of chromosome 21 (trisomy 21) and mutations in the GATA1 gene – interact to derail early blood cell development.

Researchers at Children’s Hospital of Philadelphia (CHOP) sought to better understand how the body regulates activated FVIII function, especially in interactions with other clotting factors, to inform associated pathology and therapeutic drug design.

Researchers in the Center for Computational and Genomic Medicine at Children’s Hospital of Philadelphia (CHOP) developed a new tool called the Methylation Screening Array (MSA) to better understand how changes in DNA methylation-mediated regulation of genes, rather than changes in the genes themselves, affect complex human traits like aging and disease risk. This is the first data set to dissect the two forms of DNA methylation---5-methylcytosine and 5-hydroxymethylcytosine---to associate characteristics such as cell composition, gene regulatory mechanism, and epigenetic age in diverse human cell lines and tissues.

CHOP and Penn researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating neurodegenerative disease.

Investigators at Children’s Hospital of Philadelphia, St. Jude Children’s Research Hospital, Dana Farber Cancer Institute, and the Children’s Oncology Group, unveiled for the first time that changes in certain genes affect an aggressive cancer, T-cell acute lymphoblastic leukemia, differently depending on genetic ancestry.