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.