Hosted by the Safadi Program of Excellence in Clinical and Translational Neuroscience The University of Chicago Medicine

Adeno-associated viruses (AAVs) have been given to many patients with inherited diseases, with few drugs achieving regulatory approval because of issues with durability and toxicity.  These issues arise because in general, AAVs being applied clinically show poor gene delivery efficiency to target tissues and cells of interest, requiring high doses. Also, once introduced to cells, gene expression cannot be regulated.  I will discuss ongoing work in the Davidson lab that demonstrates our progress in addressing these critical issues for the broader utility of AAVs for human therapies.

The Davidson lab is focused on genetic diseases that affect the brain. Her group studies the mechanisms by which mutant gene products contribute to disease, and why certain brain regions are more susceptible. The team employs advanced molecular methods, sequencing and imaging modalities in animal models, and uses a variety of molecular tools to further probe various hypotheses.  The lab is also engaged in the development of next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments.

Recent honors include election into the American Academy of Arts and Sciences and the National Academy of Medicine.  She is the current president of the American Society of Gene and Cell Therapy, the largest international association of gene and cell therapy research.  

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