Stem Cells, Genome Editing, and the Path to Translational Medicine

被引：103

作者：

Soldner, Frank ^{[1
]}

Jaenisch, Rudolf ^{[1
,2
]}

机构：

[1] Whitehead Inst Biomed Res, 455 Main St, Cambridge, MA 02142 USA

[2] MIT, Dept Biol, 31 Ames St, Cambridge, MA 02139 USA

来源：

CELL | 2018年 / 175卷 / 03期

关键词：

IN-VITRO; GENETIC-VARIATION; ALZHEIMERS-DISEASE; IDENTIFY COMPOUNDS; SELF-ORGANIZATION; DNA METHYLATION; BRAIN ORGANOIDS; NEURONS; MOUSE; MODEL;

D O I：

10.1016/j.cell.2018.09.010

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

The derivation of human embryonic stem cells (hESCs) and the stunning discovery that somatic cells can be reprogrammed into human induced pluripotent stem cells (hiPSCs) holds the promise to revolutionize biomedical research and regenerative medicine. In this Review, we focus on disorders of the central nervous system and explore how advances in human pluripotent stem cells (hPSCs) coincide with evolutions in genome engineering and genomic technologies to provide realistic opportunities to tackle some of the most devastating complex disorders.

引用

页码：615 / 632

页数：18

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