The Arbab Lab, based at the Rosamund Stone Zander Translational Neuroscience Center at Boston Children's Hospital and the Harvard Medical School Department of Neurology, develops precision genome editing tools to understand and treat genetic neurological disease. By combining mechanism-driven genome engineering, high-throughput functional genomics, and machine learning, we are dedicated to building novel gene-based therapeutics for diseases that remain otherwise untreatable.

Our research is focused on three interconnected frontiers: designing and optimizing therapeutic genome editing strategies for motor neuron disease, repeat expansion disorders, and intellectual and developmental disorders; engineering the next generation of programmable genome editing technologies; and developing new technologies for functional and transcriptomic profiling of patient-derived neural models, to decode the molecular basis of neurodegeneration.