Results for CRISPR

Dr. Shalem’s research focuses on translational target discovery for a range of neurodegenerative diseases. He combines technology development of large-scale CRISPR-based perturbation screens with application of such technology together with additional genomic approaches.

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.

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. 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. Tong investigates cytokine receptor signaling in normal and neoplastic hematopoietic development. She uses integrated approaches encompassing biochemistry, molecular biology, mouse models, and primary human samples to understand signaling events emanating from cytokine receptors that regulate the development of hematopoietic stem/progenitor cells.

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. 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. Castracani’s research work is focused on the most common form of X-linked sideroblastic anemia which results from mutations in the ALAS2 gene and inhibits normal heme biosynthesis.