Research
Translational genome editing
Gene-based therapies hold great promise for treatment of genetic diseases. We develop therapeutic genome editing strategies to treat genetic disorders in cells and animal models. Working in the Rosamund Stone Zander Translational Neuroscience Center, we aim to accelerate the translation of these discoveries into future therapeutics that may one day improve the lives of patients with genetic neurological diseases.
Studying genetic neurodegenerative diseases
Many neurodegenerative diseases have a genetic underpinning. The cellular processes that result from these genetic abnormalities to cause pathology are often poorly understood. Using genome editing, we modify sequences implicated in neurodegenerative disease to study them and fix them where they originate.
Predictions for precision genome editing
With the wide variety of genome editing tools available, it is difficult to predict what strategy will work best for a target of interest, even by an expert user. Empirically evaluating the genotypes and resulting phenotypes of every possible strategy is cumbersome and costly. We study genome editing tools to understand and predict the outcomes of genome editing to streamline the strategy optimization process, enable non-canonical editing of alleles, and improve precision genome editing of desired targets.
