清脆的
基因组编辑
诱导多能干细胞
Cas9
计算生物学
生物
基因组
基因
遗传学
胚胎干细胞
作者
Meeti Punetha,Sheetal Saini,Suman Chaudhary,Prem Singh Yadav,Kristin M. Whitworth,Jonathan A. Green,Dharmendra Kumar,Wilfried A. Kues
出处
期刊:Current stem cell research & therapy
[Bentham Science]
日期:2023-03-07
卷期号:19 (3): 307-315
标识
DOI:10.2174/1574888x18666230307115326
摘要
Abstract: Genome editing has enhanced our ability to understand the role of genetics in a number of diseases by facilitating the development of more precise cellular and animal models to study pathophysiological processes. These advances have shown extraordinary promise in a multitude of areas, from basic research to applied bioengineering and biomedical research. Induced pluripotent stem cells (iPSCs) are known for their high replicative capacity and are excellent targets for genetic manipulation as they can be clonally expanded from a single cell without compromising their pluripotency. Clustered, regularly interspaced short palindromic repeats (CRISPR) and CRISPR/Cas RNA-guided nucleases have rapidly become the method of choice for gene editing due to their high specificity, simplicity, low cost, and versatility. Coupling the cellular versatility of iPSCs differentiation with CRISPR/Cas9-mediated genome editing technology can be an effective experimental technique for providing new insights into the therapeutic use of this technology. However, before using these techniques for gene therapy, their therapeutic safety and efficacy following models need to be assessed. In this review, we cover the remarkable progress that has been made in the use of genome editing tools in iPSCs, their applications in disease research and gene therapy as well as the hurdles that remain in the actual implementation of CRISPR/Cas systems.
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