Human neurological diseases such as autism, schizophrenia, Alzheimer's disease and Parkinson disease are highly prevalent and cause a large financial burden to affected families. Yet, these diseases suffer from extremely limited treatment options. One new set of methods to accelerate treatment of brain diseases is to generate patient-matched neurons resembling those impacted by disease, either by using pluripotent stem cells or generating them directly from skin cells. A barrier to using these cells is that it is presently difficult to produce the correct neuronal type without also generating unwanted types. Here we propose a series of experiments to identify the best methods to produce specific neuronal types from individuals for use in studying a broad range of brain diseases. These results are also likely to aid in producing neurons or neural stem cell that can be used to repair damaged brain or spinal cord regions arising from injury or stroke.
Statement of Benefit to California:
The State of California has invested in stem cell research designed to produce valuable new disease models and to accelerate the path towards using patient matched cells for regenerative medicine. A major unsolved need in California is caused by the growing burden to State residents due to diseases that affect the brain such as autism and Alzheimer’s disease. This proposal aims to reduce that burden by developing new, efficient and cost effective methods to produce neurons of specific subtypes that match those impacted by various diseases, which is presently not possible. Success in this mission will broadly impact state-wide efforts to study disease and contribute to translational medicine aimed at diseases of the brain.