Stem cell models of neurodegenerative diseases
We aim to understand why neurons die in neurodegenerative diseases such as Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. By applying CRISPR-Cas9 genome editing to patient-derived stem cells, we generate clinically relevant human models of neurodegeneration. Using a combination of genomics, bioinformatics, and high-throughput functional screens, we interrogate these models to elucidate the regulatory networks that drive neuronal death. Our ultimate goal is to identify key disease drivers that can be used to develop therapies to halt or even reverse the relentless neuronal loss.