Investigating human disease using stem cell models

被引：187

作者：

Sterneckert, Jared L. ^{[1
]}

Reinhardt, Peter ^{[1
]}

Schoeler, Hans R. ^{[1
]}

机构：

[1] Max Planck Inst Mol Biomed, Dept Cell & Dev Biol, D-48149 Munster, Germany

来源：

NATURE REVIEWS GENETICS | 2014年 / 15卷 / 09期

关键词：

LONG QT SYNDROME; FAMILIAL ALZHEIMERS-DISEASE; HEPATOCYTE-LIKE CELLS; IPSC-DERIVED NEURONS; FRAGILE-X-SYNDROME; GENETIC CORRECTION; MOTOR-NEURONS; HOMOLOGOUS RECOMBINATION; PARKINSONS-DISEASE; FRONTOTEMPORAL DEMENTIA;

D O I：

10.1038/nrg3764

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Tractable and accurate disease models are essential for understanding disease pathogenesis and for developing new therapeutics. As stem cells are capable of self-renewal and differentiation, they are ideally suited both for generating these models and for obtaining the large quantities of cells required for drug development and transplantation therapies. Although proof of principle for the use of adult stem cells and embryonic stem cells in disease modelling has been established, induced pluripotent stem cells (iPSCs) have demonstrated the greatest utility for modelling human diseases. Furthermore, combining gene editing with iPSCs enables the generation of models of genetically complex disorders.

引用

页码：625 / 639

页数：15

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