Current RNA-based Therapeutics in Clinical Trials

Author(s): Ling-Yan Zhou, Zhou Qin, Yang-Hui Zhu, Zhi-Yao He*, Ting Xu*.

Journal Name: Current Gene Therapy

Volume 19 , Issue 3 , 2019

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Graphical Abstract:


Abstract:

Long-term research on various types of RNAs has led to further understanding of diverse mechanisms, which eventually resulted in the rapid development of RNA-based therapeutics as powerful tools in clinical disease treatment. Some of the developing RNA drugs obey the antisense mechanisms including antisense oligonucleotides, small interfering RNAs, microRNAs, small activating RNAs, and ribozymes. These types of RNAs could be utilized to inhibit/activate gene expression or change splicing to provide functional proteins. In the meantime, some others based on different mechanisms like modified messenger RNAs could replace the dysfunctional endogenous genes to manage some genetic diseases, and aptamers with special three-dimensional structures could bind to specific targets in a high-affinity manner. In addition, the recent most popular CRISPR-Cas technology, consisting of a crucial single guide RNA, could edit DNA directly to generate therapeutic effects. The desired results from recent clinical trials indicated the great potential of RNA-based drugs in the treatment of various diseases, but further studies on improving delivery materials and RNA modifications are required for the novel RNA-based drugs to translate to the clinic. This review focused on the advances and clinical studies of current RNA-based therapeutics, analyzed their challenges and prospects.

Keywords: Antisense oligonucleotide, small interfering RNA, microRNA, small activating RNA, ribozyme, aptamer, messenger RNA, single guide RNA.

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