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Current Progress in Messenger RNA-Based Gene Therapy.
Journal of Biomedical Nanotechnology ( IF 2.9 ) Pub Date : 2020-12-15 , DOI: 10.1166/jbn.2020.2961
Sibei Lei , Xueyan Zhang , Jingmei Li , Yan Gao , Jieping Wu , Xingmei Duan , Ke Men

Based on its rapid expression, simple sequence composition, low immunogenicity, and flexible modification possibilities, in vitro synthesized mRNA has demonstrated strong potential as a candidate for gene therapy. Many efforts have been made to enhance its therapeutic efficacy and safety. Profiting from the development in pathogenesis and materials science, much progress has been achieved in mRNA-based therapy studies. Many mRNA-derived therapeutics including vaccines, antibodies, cytokines, and growth factors have emerged for the treatment of diverse diseases that have multiple modes of action. Novel delivery vectors with enhanced capacity, safety, and properties have been developed to meet the demands of mRNA delivery. Advanced strategies like library screening, environment interaction, and bio-inspiration materials have been used in the investigation process and produced valuable results. In this review, we summarize and discuss recent advances in mRNA-based gene therapy studies.

中文翻译:

基于Messenger RNA的基因治疗的最新进展。

基于其快速表达,简单的序列组成,低免疫原性和灵活的修饰可能性,在体外合成的mRNA已显示出强大的潜力,可作为基因治疗的候选药物。已经进行了许多努力来增强其治疗功效和安全性。得益于发病机理和材料科学的发展,基于mRNA的治疗研究已取得许多进展。已经出现了许多源自mRNA的疗法,包括疫苗,抗体,细胞因子和生长因子,用于治疗具有多种作用方式的多种疾病。已经开发出具有增强的容量,安全性和特性的新型递送载体来满足mRNA递送的需求。研究过程中使用了图书馆筛选,环境相互作用和生物启发材料等先进策略,并产生了可贵的结果。在这篇评论中
更新日期:2020-12-16
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