Alzheimers disease in a dish: promises and challenges of human stem cell models

作者：Young Jessica E; Goldstein Lawrence S B^*

来源：Human Molecular Genetics, 2012, 21(R1): R82-R89.

DOI：10.1093/hmg/dds319

Human pluripotent stem cells can differentiate into disease-relevant cell types, which capture the unique genome of an individual patient and provide insight into pathological mechanisms of human disease. Recently, human stem cell models for Alzheimers disease (AD), the most common neurodegenerative dementia, have been described. Stem cell-derived neurons from patients with familial and sporadic AD and Downs syndrome recapitulate human disease phenotypes such as amyloid peptide production, hyperphosphorylation of tau protein and endosomal abnormalities. Treatment of human neurons with small molecules can modulate these phenotypes, demonstrating the utility of this system for drug development and screening. This review will highlight the current AD stem cell models and discuss the remaining challenges and potential future directions of this field.

• 出版日期2012-10-15