Nick Manfred

My research aims to advance treatment of and provide mechanistic insights into the pathogenesis of a rare form of childhood neurodegeneration, Batten disease. Through multi-omic approaches, I have characterized multiple mouse models of Batten disease. I now aim to ameliorate hallmarks of the disease via hematopoietic stem cell transplantation, which replaces a subset of dysfunctional brain cells (microglia) with healthy donor cells.