Our lab has pioneered the directed differentiation of human pluripotent stem cells into multiple specialized cell types of both the central and peripheral nervous system. The long-term goal is to develop a roadmap to re-create the full cellular diversity of the nervous system. In addition to cell fate, we are interested in defining the molecular pathways that control timing of human differentiation including cell fate specification, neuronal maturation and the programming and re-programming of cellular age in a dish.
One of the most intriguing applications of human pluripotent stem cells is the possibility of recreating a disease in a dish and to use such cell-based models for drug discovery. Our lab uses human iPS and ES cells for modeling human disorders of the brain and peripheral nervous system such as Parkinson's disease, familial dysautonomia, Herpes Simplex encephalitis and melanocyte related disorders including cancer.
We have a long-standing interest in developing stem cell based therapies for regenerative medicine. Our main current effort is on Parkinson's disease (PD). We have developed protocols to derive midbrain dopamine neurons from human ES and iPS cells capable of engraftment in mouse, rat and monkey models of PD. Based on such data we were awarded a consortium award from NYSTEM to pursue the first clinical application of human ES cell based dopamine neurons in PD patients.