Division of Pharmaceutics & Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, United States.
Division of Pharmaceutics & Pharmacology, College of Pharmacy, The Ohio State University, Columbus, OH 43210, United States; Department of Biomedical Engineering, The Center for Clinical and Translational Science, The Comprehensive Cancer Center, Dorothy M. Davis Heart & Lung Research Institute, Department of Radiation Oncology, The Ohio State University, Columbus, OH 43210, United States.
Adv Drug Deliv Rev. 2022 Jan;180:114042. doi: 10.1016/j.addr.2021.114042. Epub 2021 Nov 9.
RNA-binding proteins (RBPs) play an important role in RNA metabolism, regulating the stability, localization, and functional dynamics of RNAs. Alternation in the RBP-RNA network has profound implications in cellular physiology, and is related to the development and spread of cancer in certain cases. To regulate the expression of specific genes and their biological activities, various strategies have been applied to target RBPs for cancer treatments, including small-molecule inhibitors, small-interfering RNA, peptides, and aptamers. Recently, the deployment of the CRISPR-Cas9 technology has provided a new platform for RBP screening and regulation. This review summarizes the delivery systems of the CRISPR-Cas9 system and their role in RBP-based cancer therapeutics, including identification of novel RBPs and regulation of cancer-associated RBPs. The efficient delivery of the CRISPR-Cas9 system is important to the profound understanding and clinical transition of RBPs as cancer therapeutic targets.
RNA 结合蛋白（RBPs）在 RNA 代谢中发挥重要作用，调节 RNA 的稳定性、定位和功能动态。RBP-RNA 网络的改变对细胞生理学有深远的影响，在某些情况下与癌症的发展和扩散有关。为了调节特定基因的表达及其生物活性，已经应用了各种策略来靶向 RBPs 进行癌症治疗，包括小分子抑制剂、小干扰 RNA、肽和适体。最近，CRISPR-Cas9 技术的应用为 RBP 筛选和调控提供了新的平台。本综述总结了 CRISPR-Cas9 系统的递送系统及其在基于 RBP 的癌症治疗中的作用，包括鉴定新的 RBPs 和调控与癌症相关的 RBPs。CRISPR-Cas9 系统的有效递送对于深入理解和临床转化 RBPs 作为癌症治疗靶点非常重要。
