Results for Gene Editing

Dr. Lin studies RNA modifications (a.k.a "epitranscriptomics") in human diseases, including cancer. She develops and applies high-throughput sequencing strategies and transcriptome engineering technologies to study the regulation and function of RNA modifications, including A-to-I RNA editing and m6A RNA methylation.

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

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. 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. Ackermann studies diabetes (types 1 and 2) and congenital hyperinsulinism using mouse models, cell lines, and primary human tissue. She aims to identify novel pathways regulating beta cell insulin secretion, leading to innovative therapeutic strategies for these disorders. Current studies include in vivo mouse physiology, ex vivo human islet physiology, CRISPR-Cas9 gene editing, epigenetic modification, and single-cell functional genomics.

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. Bailis aims to understand how metabolism underlies immunology and disease, by controlling the biochemistry of cells and tissues. His lab does so using in vitro and in vivo CRISPR engineering of primary human and mouse immune cells, with the goal of developing diet and metabolite based therapies.