Modeling neurodevelopmental and neurodegenerative diseases using human iPSCs


April 7, 2015 - 1:00pm
NW 243
About the Speaker
Tracy Young-Pearse (Brigham Neurology)

Our understanding of the human brain has been substantially bolstered through centuries of research on the diseases that affect its function. The dysfunction of circuits in the brain important for cognition and behavior underlie two pathophysiologically and clinically distinct disorders, schizophrenia and Alzheimer’s disease. In Alzheimer’s, neurons degenerate in the aged brain which affects certain circuits, while in schizophrenia there is a defect in the development of the same or nearby circuits. Both of these prevalent disorders are intensively studied by academic and pharmaceutical labs throughout the world. However, no disease-modifying treatments exist for Alzheimer’s or schizophrenia, in part due to a lack of the fields’ understanding of these diseases. Recent genetic studies have provided clues regarding the molecular and cellular processes underlying cerebrocortical dysfunction. Furthermore, new technologies are available to address longstanding questions in degenerative and psychiatric disease in novel ways, including 1) the ability to make a variety of neuronal and glial fates from patient-derived stem cells, and 2) single cell analyses that allow for examinations of cell fate-specific phenotypes. In our lab, we employ these technologies and others to address three overarching goals central to our understanding of the developing and aging brain, as they relate to Alzheimer’s disease and schizophrenia: 1) To elucidate the physiological role of certain genes and signaling pathways linked to these disorders, 2) To determine the cell types and cell functions most affected by disease-relevant mutations, and 3) To identify and validate novel therapeutic targets for these diseases.