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Induced pluripotent stem cells and neurological disease models

Sa Cai, Ying-Shing Chan, Daisy Kwok-Yan Shum

Departments of 1Biochemistry and Physiology, and Research Centre of Heart, Brain, Hormone and Healthy Aging, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China

Abstract

The availability of human stem cells heralds a new era for in vitro cell-based modeling of neurodevelopmental and neurodegenerative diseases. Adding to the excitement is the discovery that somatic cells of patients can be reprogrammed to a pluripotent state from which neural lineage cells that carry the disease genotype can be derived. These in vitro cell-based models of neurological diseases hold promise for monitoring of disease initiation and progression, and for testing of new drug treatments on the patient-derived cells. In this review, we focus on the prospective applications of different stem cell types for disease modeling and drug screening. We also highlight how the availability of patient-specific induced pluripotent stem cells (iPS cells) offers a unique opportunity for studying and modeling human neurodevelopmental and neurodegenerative diseases in vitro and for testing small molecules or other potential therapies for these disorders. Finally, the limitations of this technology from the standpoint of reprogramming efficiency and therapeutic safety are discussed.

Key words: embryonic stem cells; induced pluripotent stem cells; neurological diseases; disease modeling; drug screening

Received: 　　Accepted:

Corresponding author: 岑国欣　　E-mail: shumdkhk@hku.hk

Citing This Article：

Sa Cai, Ying-Shing Chan, Daisy Kwok-Yan Shum. Induced pluripotent stem cells and neurological disease models. Acta Physiol Sin 2014; 66 (1): 55-66 (in Chinese with English abstract).