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The challenge and prospect of mRNA therapeutics landscape.
Biotechnology Advances ( IF 12.1 ) Pub Date : 2020-02-21 , DOI: 10.1016/j.biotechadv.2020.107534
Yuhua Weng 1 , Chunhui Li 1 , Tongren Yang 1 , Bo Hu 1 , Mengjie Zhang 1 , Shuai Guo 1 , Haihua Xiao 2 , Xing-Jie Liang 3 , Yuanyu Huang 1
Affiliation  

Messenger RNA (mRNA)-based therapeutics hold the potential to cause a major revolution in the pharmaceutical industry because they can be used for precise and individualized therapy, and enable patients to produce therapeutic proteins in their own bodies without struggling with the comprehensive manufacturing issues associated with recombinant proteins. Compared with the current therapeutics, the production of mRNA is much cost-effective, faster and more flexible because it can be easily produced by in vitro transcription, and the process is independent of mRNA sequence. Moreover, mRNA vaccines allow people to develop personalized medications based on sequencing results and/or personalized conditions rapidly. Along with the great potential from bench to bedside, technical obstacles facing mRNA pharmaceuticals are also obvious. The stability, immunogenicity, translation efficiency, and delivery are all pivotal issues need to be addressed. In the recently published research results, these issues are gradually being overcome by state-of-the-art development technologies. In this review, we describe the structural properties and modification technologies of mRNA, summarize the latest advances in developing mRNA delivery systems, review the preclinical and clinical applications, and put forward our views on the prospect and challenges of developing mRNA into a new class of drug.



中文翻译:

mRNA治疗领域的挑战和前景。

基于信使RNA(mRNA)的治疗剂有潜力在制药行业引起一场重大革命,因为它们可用于精确和个性化的治疗,并使患者能够在自己的体内产生治疗蛋白,而不会遇到与之相关的全面制造问题与重组蛋白。与目前的疗法相比,mRNA的生产具有很高的成本效益,更快,更灵活,因为它可以通过体外轻松生产转录,并且该过程与mRNA序列无关。此外,mRNA疫苗使人们能够基于测序结果和/或个性化状况快速开发个性化药物。除了从工作台到床边的巨大潜力外,mRNA药物面临的技术障碍也很明显。稳定性,免疫原性,翻译效率和递送都是需要解决的关键问题。在最近发表的研究结果中,这些问题已被最先进的开发技术逐渐克服。在这篇综述中,我们描述了mRNA的结构特性和修饰技术,总结了开发mRNA递送系统的最新进展,回顾了临床前和临床应用,

更新日期:2020-02-21
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