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，我们将回顾CRISPR-Cas系统及其最新发展，重点是对人类细胞的应用。我们还将讨论如何基于CRISPR的不同策略来实现特定的基因组工程目标。然后，我们回顾了如何在体外人类干细胞基因组工程中使用不同的CRISPR工具，涵盖多能（iPSC / ESC）和成体干细胞领域，尤其是3D类器官培养。最后，我们讨论了用于治疗用途的人类干细胞基于CRISPR的基因组编辑相关的进展和挑战。