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Yanhong Shi

Beckman Research Institute of City of Hope

Yanhong Shi is Herbert Horvitz Professor in Neuroscience and Director of Division of Stem Cell Biology Research at Beckman Research Institute of City of Hope. She earned her Ph.D. degree from Northwestern University. Upon graduation, she went to the Salk Institute for postdoctoral training, where she studied nuclear receptors in neural stem cells and neurogenesis. Her lab at Beckman Research Institute of City of Hope focuses on human iPSC-based disease modeling, drug discovery, and cell therapy development for neurological diseases. 

Human IPSC-Based Disease Modeling and Therapeutic Development

Abstract:

The iPSC technology has provided great hope for developing cell therapies, in addition to serving as a platform for disease modeling and drug discovery. We have used the human iPSC platform to model neurological disorders and develop cell therapies for neurological diseases. For example, we have established stem cell therapy candidates for Canavan disease (CD), a devastating neurological disease that has neither a cure nor a standard treatment. We established a cGMP-compatible manufacturing process for human iPSC derivation and subsequent differentiation and genetic modification. We further tested the human iPSC-derived cellular products in a Canavan disease mouse model and demonstrated robust efficacy and preliminary safety of the cellular products. This study could provide an effective therapeutic approach, and eventually lead to a cure for Canavan disease, and other related neurological diseases.