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.
Category Archive: Raymond G. Perelman Center for Cellular & Molecular Therapeutics
Researchers gained new insights into the heart problems that are the second leading cause of death in patients with Huntington’s disease (HD). An incurable, inherited disease with progressive loss of brain cells and motor function, HD occurs when a defective gene produces repeated copies of a protein called huntingtin, or HTT. The mutant HTT (mHTT) protein disrupts multiple fundamental cellular processes along the mTORC1 pathway that promotes cell growth and metabolism. The study team described how decreased mTORC1 activity contributed to the development of heart disease with stress in mouse models of HD. By restoring cardiac mTORC1 activity, the researchers improved the animals’ heart function and survival over the course of the study.
In the halls and history of Children’s Hospital of Philadelphia and its Research Institute, you don’t have to search far to find role models who thrive in science fields that have been, historically, underrepresented by women.
September marks National Childhood Cancer Awareness Month, and this year at Children’s Hospital of Philadelphia, we kick-started the commemorative period on the heels of exciting news about breakthroughs in pediatric cancer immunotherapy research. Oncology investigators at CHOP also got a big boost in research funding from Hyundai’s nonprofit organization, Hope on Wheels. And that’s only the beginning: Since September marks the return of the football season, we’re thrilled to share the latest headlines on how the National Football League (NFL) is helping to drive concussion research.
This week in the news, those sorts of everyday queries – whether they’re about how to breastfeed, why parents should follow a vaccine schedule, or how mechanical circulatory support devices work – led to exciting headline-making stories.
One of the things that is so exciting about research at CHOP is that our researchers are working to improve the health and lives of children in such a broad range of ways.
Although the year is coming to a close, the research achievements at The Children’s Hospital of Philadelphia in 2015 remain enduring contributions to pediatric health.
Scientists at The Children’s Hospital of Philadelphia are comparing two molecular therapy techniques for Dystonia — RNA interference (RNAi) and antisense oligonucleotides (ASOs).
Researchers in the Center for Cellular and Molecular Therapeutics (CCMT) at The Children’s Hospital of Philadelphia suggest that an intricate pathway crucial to the development of Huntington’s disease (HD) rests on a “biological teeter-totter” that when carefully balanced could help to control this devastating neurodegenerative disorder.
Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1.