RNA Therapeutics - Research and Clinical Advancements

Rundong Feng; Suryaji Patil; Xin Zhao; Zhiping Miao; Airong Qian

doi:10.3389/fmolb.2021.710738

RNA Therapeutics - Research and Clinical Advancements

Front Mol Biosci. 2021 Sep 22:8:710738. doi: 10.3389/fmolb.2021.710738. eCollection 2021.

Authors

Rundong Feng¹, Suryaji Patil², Xin Zhao³, Zhiping Miao², Airong Qian²

Affiliations

¹ Shaanxi Institute for Food and Drug Control, Xi'an, China.
² Lab for Bone Metabolism, Xi'an Key Laboratory of Special Medicine and Health Engineering, Key Lab for Space Biosciences and Biotechnology, Research Center for Special Medicine and Health Systems Engineering, NPU-UAB Joint Laboratory for Bone Metabolism, School of Life Sciences, Northwestern Polytechnical University, Xi'an, China.
³ School of Pharmacy, Shaanxi Institute of International Trade & Commerce, Xi'an, China.

Abstract

RNA therapeutics involve the use of coding RNA such as mRNA as well as non-coding RNAs such as small interfering RNAs (siRNA), antisense oligonucleotides (ASO) to target mRNA, aptamers, ribozymes, and clustered regularly interspaced short palindromic repeats-CRISPR-associated (CRISPR/Cas) endonuclease to target proteins and DNA. Due to their diverse targeting ability and research in RNA modification and delivery systems, RNA-based formulations have emerged as suitable treatment options for many diseases. Therefore, in this article, we have summarized different RNA therapeutics, their targeting strategies, and clinical progress for various diseases as well as limitations; so that it might help researchers formulate new and advanced RNA therapeutics for various diseases. Additionally, U.S. Food and Drug Administration (USFDA)-approved RNA-based therapeutics have also been discussed.

Keywords: CRISPR-Cas; FDA approved; RNA-based therapy; antisense oligonucleotides; aptamers; clinical trial; siRNA.

Publication types

Review