Results for Genome Editing

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. Cardinale's research is focused on understanding the mechanisms of gene expression and gene regulation in autoimmune diseases, including inflammatory bowel disease, type 1 diabetes, and systemic sclerosis. He uses data from large-scale genomic studies to identify disease-causing genetic variants and functionally explore the target genes of those variants.

Dr. Peranteau is an attending surgeon in the Division of Pediatric General, Thoracic and Fetal Surgery and an associate professor of Surgery at CHOP. His research interest is in fetal development as it relates to the potential understanding of and fetal intervention for congenital anomalies.

Dr. Choi's research focuses on the role of RNA-binding proteins in the regulation of alternative splicing and how mutations in these factors contribute to cancer. He uses a combination of genetically-engineered models and high-throughput approaches to better understand how alternative splicing influences cellular function and to identify potential opportunities for therapeutic intervention.

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.