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Deliver the promise: RNAs as a new class of molecular entities for therapy and vaccination.
Yu, Ai-Ming; Tu, Mei-Juan.
  • Yu AM; Department of Biochemistry and Molecular Medicine, UC Davis School of Medicine, Sacramento, CA 95817, USA. Electronic address: aimyu@ucdavis.edu.
  • Tu MJ; Department of Biochemistry and Molecular Medicine, UC Davis School of Medicine, Sacramento, CA 95817, USA.
Pharmacol Ther ; 230: 107967, 2022 02.
Article in English | MEDLINE | ID: covidwho-1757736
ABSTRACT
The concepts of developing RNAs as new molecular entities for therapies have arisen again and again since the discoveries of antisense RNAs, direct RNA-protein interactions, functional noncoding RNAs, and RNA-directed gene editing. The feasibility was demonstrated with the development and utilization of synthetic RNA agents to selectively control target gene expression, modulate protein functions or alter the genome to manage diseases. Rather, RNAs are labile to degradation and cannot cross cell membrane barriers, making it hard to develop RNA medications. With the development of viable RNA technologies, such as chemistry and pharmaceutics, eight antisense oligonucleotides (ASOs) (fomivirsen, mipomersen, eteplirsen, nusinersen, inotersen, golodirsen, viltolarsen and casimersen), one aptamer (pegaptanib), and three small interfering RNAs (siRNAs) (patisiran, givosiran and lumasiran) have been approved by the United States Food and Drug Administration (FDA) for therapies, and two mRNA vaccines (BNT162b2 and mRNA-1273) under Emergency Use Authorization for the prevention of COVID-19. Therefore, RNAs have become a great addition to small molecules, proteins/antibodies, and cell-based modalities to improve the public health. In this article, we first summarize the general characteristics of therapeutic RNA agents, including chemistry, common delivery strategies, mechanisms of actions, and safety. By overviewing individual RNA medications and vaccines approved by the FDA and some agents under development, we illustrate the unique compositions and pharmacological actions of RNA products. A new era of RNA research and development will likely lead to commercialization of more RNA agents for medical use, expanding the range of therapeutic targets and increasing the diversity of molecular modalities.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: COVID-19 / BNT162 Vaccine Type of study: Randomized controlled trials Topics: Vaccines Limits: Humans Country/Region as subject: North America Language: English Journal: Pharmacol Ther Year: 2022 Document Type: Article

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Full text: Available Collection: International databases Database: MEDLINE Main subject: COVID-19 / BNT162 Vaccine Type of study: Randomized controlled trials Topics: Vaccines Limits: Humans Country/Region as subject: North America Language: English Journal: Pharmacol Ther Year: 2022 Document Type: Article