Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system represent two instruments of basic and translational research, which both allow to acquire deep insight about the molecular bases of many diseases but also to develop pharmacological research. This review is focused to draw up the latest technique of gene editing applied on hiPSCs, exploiting some of the genetic manipulation directed to the discovery of innovative therapeutic strategies. There are many expediencies provided by the use of hiPSCs, which can represent a disease model clinically relevant and predictive, with a great potential if associated to CRISPR/Cas9 technology, a gene editing tool powered by ease and precision never seen before. Here, we describe the possible applications of CRISPR/Cas9 to hiPSCs: from drug development to drug screening and from gene therapy to the induction of the immunological response to specific virus infection, such as HIV and SARS-Cov-2.

中文翻译：

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CRISPR/Cas9 在人类诱导多能干细胞中的应用：从基因编辑到药物发现。


人诱导多能干细胞（hiPSC）和CRISPR/Cas9基因编辑系统代表了基础和转化研究的两种工具，既可以深入了解许多疾病的分子基础，又可以开展药理学研究。本综述的重点是制定应用于 hiPSC 的最新基因编辑技术，利用一些基因操作来发现创新的治疗策略。 hiPSC 的使用提供了许多便利，它可以代表临床相关和预测的疾病模型，如果与 CRISPR/Cas9 技术（一种前所未有的简便性和精确性的基因编辑工具）相关联，则具有巨大的潜力。在这里，我们描述了 CRISPR/Cas9 在 hiPSC 上的可能应用：从药物开发到药物筛选，从基因治疗到诱导对特定病毒感染（如 HIV 和 SARS-Cov-2）的免疫反应。