RNA therapy: rich history, various applications and unlimited future prospects

• Published in: Experimental & molecular medicine Vol. 54; no. 4; pp. 455 - 465
• Main Author: Kim, Young-Kook
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.04.2022; Springer Nature B.V; 생화학분자생물학회
• Subjects: 38/89; 631/154; 692/308/153; 692/699; Biomedical and Life Sciences; Biomedicine; Coronaviruses; COVID-19; COVID-19 vaccines; Drug delivery; Drug delivery systems; Medical Biochemistry; Medical research; Medical treatment; Molecular Medicine; mRNA; Rare diseases; Review; Review Article; Reviews; Stem Cells; Vaccines; 생화학
• ISSN: 2092-6413; 1226-3613; 2092-6413
• DOI: 10.1038/s12276-022-00757-5

RNA therapy refers to the treatment or prevention of diseases using RNA-based molecules. The recent advent of a series of effective messenger RNA-based vaccines in response to the COVID-19 pandemic has reignited research interest in RNA therapy. Based on the accumulated results of long-term research in the field of RNA therapy spanning several decades, therapeutic agents for various diseases are being rapidly developed. These therapeutics tend to target diseases that cannot be treated with other conventional drug groups, and several clinical studies are underway for a variety of RNA-based therapeutics against various incurable diseases. This review describes the history of several important discoveries in RNA biology and their impact on key developments in RNA therapy as well as the advantages of RNA therapy. In addition, it describes the action mechanisms and examples of drugs approved for RNA therapy. Finally, this review discusses methods for RNA drug delivery to target organs and cells. Given that RNA therapy is expected to advance and play an integral role in the development of novel therapeutic agents for human diseases in the future, this review is designed to offer an updated reference point for researchers in this field. RNA therapies: optimising drug delivery for treating the untreatable RNA-based therapies should improve the lives of many people affected by difficult-to-treat diseases, provided novel drug delivery methods are fully explored and optimized. Young-Kook Kim at Chonnam National University Medical School in Hwasun, Korea, reviewed the current status of RNA therapies, particularly given recent successes in developing messenger RNA vaccines for COVID-19. RNA therapeutics work by manipulating the expression and activity of specific target molecules, providing the means to treat diseases that do not respond to conventional drug types. Such therapies can be tweaked to cover a wide range of different forms of RNA and protein, and could open the way to personalized medicines and treatments for rare diseases. However, RNA-based drugs are larger molecules than other therapeutics, making targeted delivery within the body more difficult. Kim suggests that ensuring effective RNA drug delivery should be a paramount focus for future research.