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We use human cellular models to understanding and treat neurological disease. These diseases are historically studied using either immortalized cell lines or animal models, and while these approaches have been invaluable, concerns about the translatability of results persist. In recognition of this, human inducible pluripotent stem cell (iPSC)-derived models are a key solution, and my program specializes in the development glial-related iPSC model systems, such as for microglia, ependymal cells and Schwann cells.

Critically, we study these specific cell populations using iPSCs in the context of defined glial-related diseases, with a focus on monogenic conditions, where patients with genetic variants are already identified and characterized. We generate iPSCs from these patients and use these models for mechanistic research and drug development with a focus on testing delivery methods that are well tolerated with flexible cargo loading, such as nanoparticles.