College of Veterinary Sciences, Lala Lajpat Rai University of Veterinary and Animal Science (LUVAS), Hisar, Haryana,India.
Curr Top Med Chem. 2021;21(32):2851-2855. doi: 10.2174/1568026621666211118095451.
In this era, RNA molecules have provided a unique opportunity to researchers all over the world for expanding their range of targets in the development of drugs. Due to the unique pharmacological as well as physicochemical characteristics of different RNA molecules such as aptamers, small interfering RNAs (siRNA), antisense oligonucleotides (ASO) and guide RNAs (gRNA), they have emerged recently as a new class of drugs. They are used for selective action on proteins and genes that were not possible to target by conventional drug molecules. These RNA molecules like guide RNAs are also components of novel gene editing mechanisms which can modify the genome nearly in all cells. Vaccines based on RNA molecules have also provided a promising alternative to conventional live attenuated vaccines. RNA based vaccines have high potency, can be rapidly developed, and have potential for manufacturing at a cheaper rate and safe administration. However, the application of these RNAs has been restricted by the high instability and inefficient in vivo delivery. Technological advancement needs to overcome these issues so that RNA based drugs targeting several diseases can be developed. This article emphasizes the potential of RNA based drugs and the major barriers associated with the development of RNA therapeutics. Additionally, the role of RNA based vaccines and their challenges in advancing this promising vaccine platform for the prevention of infectious diseases have been discussed.
在这个时代，RNA 分子为世界各地的研究人员提供了一个独特的机会，扩大了他们在药物开发中目标的范围。由于不同 RNA 分子（如适体、小干扰 RNA（siRNA）、反义寡核苷酸（ASO）和指导 RNA（gRNA））具有独特的药理学和物理化学特性，它们最近已成为一类新的药物。它们用于选择性作用于蛋白质和基因，而这些蛋白质和基因是传统药物分子无法靶向的。这些 RNA 分子，如指导 RNA，也是新型基因编辑机制的组成部分，几乎可以在所有细胞中修饰基因组。基于 RNA 分子的疫苗也为传统减毒活疫苗提供了有希望的替代方案。基于 RNA 的疫苗具有高效力、可快速开发以及以更低廉的价格和安全的方式进行生产的潜力。然而，这些 RNA 的应用受到其高度不稳定性和体内递送效率低下的限制。技术进步需要克服这些问题，以便能够开发针对多种疾病的基于 RNA 的药物。本文强调了基于 RNA 的药物的潜力，以及与 RNA 治疗药物开发相关的主要障碍。此外，还讨论了基于 RNA 的疫苗的作用及其在推进这一有前途的传染病预防疫苗平台方面的挑战。
