Department of Biomedicine and Prevention, University of Rome "Tor Vergata", Rome, Italy.
Hum Genomics. 2020 Jun 26;14(1):25. doi: 10.1186/s40246-020-00276-2.
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.
人类诱导多能干细胞（hiPSCs）和 CRISPR/Cas9 基因编辑系统代表了基础和转化研究的两种工具，它们都可以深入了解许多疾病的分子基础，也可以开发药理学研究。本综述的重点是制定最新的基因编辑技术应用于 hiPSCs，利用一些遗传操作来发现创新的治疗策略。hiPSCs 的应用有很多优势，它可以作为一种具有临床相关性和预测性的疾病模型，与 CRISPR/Cas9 技术结合具有巨大的潜力，这是一种以前从未见过的具有简便性和精确性的基因编辑工具。在这里，我们描述了 CRISPR/Cas9 在 hiPSCs 中的可能应用：从药物开发到药物筛选，从基因治疗到诱导针对特定病毒感染（如 HIV 和 SARS-CoV-2）的免疫反应。
