College of Animal Science, Nanjing Agricultural University, Nanjing, China.
Parkinson's Institute and Clinical Center, Sunnyvale, CA, United States.
Curr Stem Cell Res Ther. 2018;13(4):243-251. doi: 10.2174/1574888X13666180214124800.
Induced pluripotent stem cells hold tremendous potential for biological and therapeutic applications. The development of efficient technologies for targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9) system is recently recognized as a powerful tool for editing DNA at specific loci.
The ease of use of the CRISPR-Cas9 technology will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. More recently, this system was modified to repress (CRISPR interference, CRISPRi) or activate (CRISPR activation, CRISPRa) gene expression without alterations in the DNA, which amplified the scope of applications of CRISPR systems for stem cell biology.
Here, we highlight latest advances of CRISPR-associated applications in human pluripotent stem cells. The challenges and future prospects of CRISPR-based systems for human research are also discussed.
诱导多能干细胞在生物和治疗应用方面具有巨大的潜力。开发针对疾病模型中干细胞的靶向基因组修饰的高效技术是充分利用干细胞的前提。最近，被称为成簇规律间隔短回文重复（CRISPR）相关蛋白 9（Cas9）系统的基因组编辑革命性技术被认为是在特定基因座编辑 DNA 的强大工具。
CRISPR-Cas9 技术的易用性将使我们能够通过细胞和动物模型来提高对疾病过程中基因组变异的理解。最近，该系统经过改良，可以在不改变 DNA 的情况下抑制（CRISPR 干扰，CRISPRi）或激活（CRISPR 激活，CRISPRa）基因表达，从而扩大了 CRISPR 系统在干细胞生物学中的应用范围。
在这里，我们重点介绍 CRISPR 相关应用在人类多能干细胞中的最新进展。还讨论了基于 CRISPR 的系统用于人类研究的挑战和未来前景。
