Duan Zhijian, Yu Ai-Ming
a Department of Biochemistry & Molecular Medicine , Comprehensive Cancer Center, UC Davis School of Medicine , Sacramento , CA , USA.
Bioengineered. 2016 Nov;7(6):411-417. doi: 10.1080/21655979.2016.1207011. Epub 2016 Jul 14.
Non-coding RNAs (ncRNAs) including microRNAs (miRNAs) and small interfering RNAs (siRNAs) are important players in the control of gene regulation and represent novel promising therapeutic targets or agents for the treatment of various diseases. While synthetic ncRNAs are predominately utilized, the effects of excessive artificial modifications on higher-order structures, activities and toxicities of ncRNAs remain uncertain. Inspired by recombinant protein technology allowing large-scale bioengineering of proteins for research and therapy, efforts have been made to develop practical and effective means to bioengineer ncRNA agents. The fermentation-based approaches shall offer biological ncRNA agents with natural modifications and proper folding critical for ncRNA structure, function and safety. In this article, we will summarize current recombinant RNA platforms to the production of ncRNA agents including siRNAs and miRNAs. The applications of bioengineered ncRNA agents for basic research and potential therapeutics are also discussed.
包括微小RNA（miRNA）和小干扰RNA（siRNA）在内的非编码RNA（ncRNA）是基因调控控制中的重要参与者，并且是治疗各种疾病的新型有前景的治疗靶点或药物。虽然主要使用合成ncRNA，但过度人工修饰对ncRNA的高级结构、活性和毒性的影响仍不确定。受允许对蛋白质进行大规模生物工程用于研究和治疗的重组蛋白技术的启发，人们已努力开发实用且有效的方法来对ncRNA药物进行生物工程改造。基于发酵的方法应能提供具有天然修饰且折叠正确的生物ncRNA药物，这对ncRNA的结构、功能和安全性至关重要。在本文中，我们将总结当前用于生产包括siRNA和miRNA在内的ncRNA药物的重组RNA平台。还将讨论生物工程改造的ncRNA药物在基础研究和潜在治疗方面的应用。
