Journal
FRONTIERS IN MOLECULAR BIOSCIENCES
Volume 8, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fmolb.2021.710738
Keywords
RNA-based therapy; siRNA; antisense oligonucleotides; aptamers; CRISPR-Cas; clinical trial; FDA approved
Categories
Funding
- National Natural Science Foundation of China [82072106]
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RNA therapeutics involve using coding RNA such as mRNA, non-coding RNAs like siRNA and ASO to target proteins and DNA. With research in RNA modification and delivery systems, RNA-based formulations have emerged as suitable treatment options for many diseases. This article summarizes different RNA therapeutics, targeting strategies, clinical progress, and limitations, aiming to help researchers create new and advanced RNA therapeutics for various diseases.
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.
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