Dr. Pavani manages the human stem cell model development of ENDD. She is also co-Director – with Dr. French – of the Human Pluripotent Stem Cell Core at The Children’s Hospital of Philadelphia (CHOP) and a Research Assistant Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine, University of Pennsylvania.
Dr. Pavani has extensive knowledge in gene editing technologies -including CRISPR/Cas9, base editors and site-specific recombinases- and has developed several editing approaches in cell lines, pluripotent stem cells, and primary hematopoietic cells. Her skills encompass a wide range of molecular and biochemical techniques to study how mutations affect gene and protein functions and lead to pathological conditions.
She has research experience in the areas of gene and cell therapy, hematology, and pluripotent stem cell biology. Her primary research focus is hematopoietic progenitors and red cell biology, though as Director of the Human Pluripotent Stem Cell Core she has been centrally involved in a host of significant scientific collaborations.
At ENDD, Dr. Pavani oversees human model development including induced pluripotent stem cell generation, characterization, and quality control; differentiation to the appropriate germ layer and derivative lineage, including hematopoietic and neuronal; and gene editing. The ability to edit genes using CRISPR/Cas9 technology has been a game-changer in the field and is being utilized to correct or introduce pathologic variants in established pluripotent stem cell lines for genotype-phenotype studies.
