Dr. Davidson is Director of The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, the Chief Scientific Strategy Officer, and holds the Arthur V. Meigs Chair in Pediatrics at the Children’s Hospital of Philadelphia. She is also Professor of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania.
Professor Davidson’s research is focused on inherited genetic diseases that cause central nervous system dysfunction, with a focus on recessive, childhood onset neurodegenerative disease, in particular the lysosomal storage diseases such as the Mucopolysaccharidoses and Batten’s disease. Additionally, a portion of her research focuses on dominant genetic diseases including the CAG repeat disorders (Huntington’s disease (HD) and Spinocerebellar ataxia) and understanding how noncoding RNAs participate in neural development and neurodegenerative diseases processes. Her research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop gene and small molecule-based medicines for therapy. In recent work, she demonstrated that the application of recombinant viral vectors to animal models of storage disease reversed CNS deficits.
For her research and efforts in gene therapy, she has received numerous awards and accolades, including the 2009 Mathilde Solowey Award (NIH), the University of Iowa Innovator Award (2012), elected to the American Academy of Arts and Sciences (2017), National Academy of Medicine (2019), and Vice President of the Board of Directors for the American Society of Gene and Cell Therapy (current). Dr. Davidson is a co-founder of Spark Therapeutics, and Talee Bio, and has served on the Scientific Advisory Boards of Sarepta Therapeutics, Homology Medicines, Prevail Therapeutics and Intellia Therapeutics.