The emergence of highly versatile genome-modifying technologies – which can be used to correct the mutations underlying many genetic disorders – has driven a revolution in biomedical research.
Our group aims to harness these and other emerging tools to develop corrective gene therapies for neurodegenerative disorders – including amyotrophic lateral sclerosis (ALS) and Alzheimer’s and Huntington’s disease. Neurodegeneration, in particular, is the leading cause of disability in the aging population and predicted by the World Health Organization to surpass cancer and become the second leading cause of death worldwide.
In addition to leveraging genome editing to treat neurological disorders, we use methods for genome-scale engineering to identify modifiers of neurodegeneration that can shed light on the mechanisms of disease and spur the development of new therapeutics. We also use molecular engineering to increase the safety of genome-editing technologies, and develop methods to curate the specificity of these tools within the nervous system.