Department of Molecular Virology, Immunology and Medical Genetics, Comprehensive Cancer Center, The Ohio State University , Columbus, OH , USA.
Courant Institute of Mathematical Sciences, New York University , New York, NY , USA.
Front Bioeng Biotechnol. 2014 Dec 11;2:65. doi: 10.3389/fbioe.2014.00065. eCollection 2014.
The use of synthetic non-coding RNAs for post-transcriptional regulation of gene expression has not only become a standard laboratory tool for gene functional studies but it has also opened up new perspectives in the design of new and potentially promising therapeutic strategies. Bioinformatics has provided researchers with a variety of tools for the design, the analysis, and the evaluation of RNAi agents such as small-interfering RNA (siRNA), short-hairpin RNA (shRNA), artificial microRNA (a-miR), and microRNA sponges. More recently, a new system for genome engineering based on the bacterial CRISPR-Cas9 system (Clustered Regularly Interspaced Short Palindromic Repeats), was shown to have the potential to also regulate gene expression at both transcriptional and post-transcriptional level in a more specific way. In this mini review, we present RNAi and CRISPRi design principles and discuss the advantages and limitations of the current design approaches.
利用合成非编码 RNA 进行基因表达的转录后调控不仅成为基因功能研究的标准实验室工具，而且为新的、有潜在前景的治疗策略的设计开辟了新的视角。生物信息学为研究人员提供了多种工具，用于设计、分析和评估 RNAi 试剂，如小干扰 RNA（siRNA）、短发夹 RNA（shRNA）、人工 microRNA（a-miR）和 microRNA 海绵。最近，一种基于细菌 CRISPR-Cas9 系统（Clustered Regularly Interspaced Short Palindromic Repeats）的基因组工程新系统被证明也有可能更特异性地在转录和转录后水平上调节基因表达。在这篇迷你综述中，我们介绍了 RNAi 和 CRISPRi 的设计原则，并讨论了当前设计方法的优缺点。
