Abstract

Investigation of Neurodegenerative Disease Utilizing Patient-Specific iPS cells

Keiko Muguruma, Research Specialist, RIKEN

Recent advances in the techniques that differentiate induced pluripotent stem cells (iPSCs) into specific types of cells enabled us to establish in vitro disease models from the patient-derived iPSCs. The advantage of the model utilizing disease-specific iPSC is that it is able to generate a large number of cells required for high-throughput screening. The patient-derived iPSCs are expected to recapitulate the disease-specific pathogenesis and physiology in vitro. We developed 3D culture systems using human pluripotent stem cells, which would promote the research on the construction of complex brain regions such as the cerebral cortex and the cerebellum. Recently we have succeeded in generation of spinocerebellar ataxia (SCA) patient-derived Purkinje cells by combining the iPSC technology and the self-organizing stem cell 3D culture technology. We have constructed an in vitro disease model recapitulating both ontogenesis and pathogenesis for SCA. Here we will talk about approaches for intractable diseases utilizing patient-specific iPSCs.