Dr. Davidson's research is focused on understanding the molecular basis for neurodegeneration in inherited brain diseases that cause central nervous system dysfunction, and then applying that knowledge toward the development of therapies. Her work extends from identifying the pathological consequences of these mutations, including splicing changes, to using cell and animal model systems to query the relevance of those alterations.

Dr. Davidson also studies how noncoding RNAs participate in neural development and neurodegenerative diseases processes. Her research on childhood onset neurodegenerative diseases includes lysosomal storage diseases such as the mucopolysaccharidoses and Batten's disease, and dominant genetic diseases such as the CAG repeat disorders Huntington’s disease and spinocerebellar ataxia. By gaining new insights into the biochemistry and cell biology of proteins deficient in these disorders, Dr. Davidson and her lab are developing gene and small molecule-based medicines for therapy.

In recent work, Dr. Davidson demonstrated that the application of recombinant viral vectors to animal models of storage disease reversed central nervous system deficits. To approach therapies for dominant disorders, her laboratory developed and tested gene silencing and CrispR/Cas9 editing strategies for reducing expression of the disease gene in patient cells and in mice models. The Davidson lab, along with colleagues experienced in gene-based medicine delivery to humans, are advancing these promising preclinical studies to clinical trials in patients. Dr. Davidson also is a co-founder of Spark Therapeutics and Talee Bio, and she serves on the scientific advisory boards of Sarepta Therapeutics and Intellia Therapeutics.

BS, Nebraska Weslyan University, Lincoln, Neb. (Biology), 1981

PhD, University of Michigan, Ann Arbor, Mich. (Biological Chemistry), 1987

Postdoctoral Fellow, University of Michigan, Ann Arbor, Mich. (Molecular Genetics), 1990

Chief Scientific Strategy Officer

Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics

Director, Research Vector Core

Arthur V. Meigs Chair in Pediatrics at Children’s Hospital of Philadelphia

Professor of Pathology and Laboratory Medicine

American Association for the Advancement of Science, 1994-

American Society for Neuroscience, 1996-

American Society of Gene and Cell Therapy, 1997-

American Society for Microbiology, 1998-

American Society for Biochemistry and Molecular Biology, 2006-

Royal Society of Medicine, 2014-

American Academy of Arts and Sciences, 2017-

The RNA Society, 2017-

College of Physicians, Philadelphia Pennsylvania, 2018-

American Society of Human Genetics, 2018-

Mathilde Solowey Award, National Institutes of Health, 2009

S.J. DeArmond Lecture, American Association of Neuropathologists, May 2011

Presidential Lecture, University of Iowa, 2011

Distinguished Alumni Lecture, University of Michigan, 2011

Finalist, Pioneer Hi-Bred Iowa Women of Innovation Awards, Technology Association of Iowa, 2011

Carver College of Medicine Faculty Service Award, University of Iowa, 2012

University of Iowa Innovator Award, 2012

Chair, Electorate Nominating Committee, Medical Sciences Section, American Association for the Advancement of Science, 2014

Appointed to the National Advisory Council, National Institute of Neurological Disorders and Stroke, 2014-2018

Appointed to Scientific Advisory Boards of the Huntington Study Group and the Medical Research Advisory Board of the National Ataxia Foundation, 2015

The Leslie Gehry Brenner Prize for Innovation in Science, Hereditary Disease Foundation, 2015


Elected to the American Academy of Arts and Sciences, 2017

F.E. Bennett Memorial Lectureship Award, American Neurological Association, 2018

Member, Electorate Nominating Committee for Section V (Neuroscience), AAAS, 2018

Election to National Academy of Medicine, 2019

Chair, Gordon Research Conference on Lysosomal Storage Disease, 2019


Frontiers in Neuroscience Plenary Session presenter, AAN, 2019