Results for Therapeutic Modeling

Dr. French came to CHOP in 2008 to establish the Human Pluripotent Stem Cell Core in the Center for Cellular and Molecular Therapeutics. She is an internationally recognized researcher involved in multi-investigator teams that utilize pluripotent stem cells for modeling human disease to study mechanism, development, and establish new therapeutic modalities.

Dr. De Leon-Crutchlow’s translational research program focuses on examining the pathophysiology of disorders of insulin regulation, identifying novel therapeutic targets, and developing new therapies for these conditions. The program approach includes patient-oriented research and bench research employing mouse models and primary islet cultures.

Dr. Falk is a Clinical Geneticist who serves as executive director of the Mitochondrial Medicine Frontier Program. Her translational research lab investigates the causes and global metabolic consequences of mitochondrial disease, as well as targeted therapies, in C. elegans, zebrafish, mouse, and human tissue models of genetic-based respiratory chain dysfunction.

Dr. Gonzalez-Alegre's long-range research goal is to advance the application of precision medicine in the neurology clinic. His research focus revolves around genetic disorders that affect the brain, spanning from the diagnosis of novel genetic disease in the clinic to the identification of novel molecular targets using disease models and the design of early-phase human clinical trials.

Dr. Smith is a laboratory animal medicine veterinarian. As Technical Director of the CCMT Comparative Medicine Services Core, he helps guide promising therapeutics and devices from discovery and developmental studies through nonclinical studies.

Dr. De Raedt researches pediatric high grade glioma development and aims to understand the involvement of crucial pathways. He investigates pathway interaction, and explores ways to develop therapies through analyzing human tumors, performing cellular studies, and developing accurate mouse models. This allows Dr. De Raedt and his team to perform novel pre-clinical studies that can lead to clinical trials.

Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.

Dr. Weber is developing approaches to enhance CAR-T cell therapies for pediatric cancer by reprogramming T cells with improved durability and exhaustion resistance. His work will uncover molecular mechanisms that promote CAR-T cell exhaustion and identify new targets for therapeutic intervention.

Dr. Leavens uses stem cell-derived beta cells to model pediatric beta cell disease, including monogenic diabetes and congenital hyperinsulinism. The goal of her studies is to better understand and develop novel therapeutics to treat these diseases.

Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.