CRISPR-Cas 工具及其在人类干细胞和类器官基因工程中的应用。
CRISPR-Cas Tools and Their Application in Genetic Engineering of Human Stem Cells and Organoids.
机构信息
Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences, and University Medical Center, Utrecht, the Netherlands; Oncode Institute, Utrecht, the Netherlands.
Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences, and University Medical Center, Utrecht, the Netherlands; Oncode Institute, Utrecht, the Netherlands; The Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands.
出版信息
Cell Stem Cell. 2020 Nov 5;27(5):705-731. doi: 10.1016/j.stem.2020.10.014.
CRISPR-Cas technology has revolutionized biological research and holds great therapeutic potential. Here, we review CRISPR-Cas systems and their latest developments with an emphasis on application to human cells. We also discuss how different CRISPR-based strategies can be used to accomplish a particular genome engineering goal. We then review how different CRISPR tools have been used in genome engineering of human stem cells in vitro, covering both the pluripotent (iPSC/ESC) and somatic adult stem cell fields and, in particular, 3D organoid cultures. Finally, we discuss the progress and challenges associated with CRISPR-based genome editing of human stem cells for therapeutic use.
CRISPR-Cas 技术已经彻底改变了生物学研究，并具有巨大的治疗潜力。在这里，我们回顾了 CRISPR-Cas 系统及其最新进展，重点介绍了其在人类细胞中的应用。我们还讨论了如何使用不同的基于 CRISPR 的策略来实现特定的基因组工程目标。然后，我们回顾了不同的 CRISPR 工具在人类干细胞的基因组工程中的应用，涵盖了多能（iPSC/ESC）和成体干细胞领域，特别是 3D 类器官培养。最后，我们讨论了基于 CRISPR 的基因组编辑用于人类干细胞治疗的相关进展和挑战。
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