RNA has sparked a revolution in modern medicine, with the potential to transform the way we treat diseases. Recent regulatory approvals, hundreds of new clinical trials, the emergence of CRISPR gene editing, and the effectiveness of mRNA vaccines in dramatic response to the COVID-19 pandemic have converged to create tremendous momentum and expectation. However, challenges with this relatively new class of drugs persist and require specialized knowledge and expertise to overcome. This Review explores shared strategies for developing RNA drug platforms, including layering technologies, addressing common biases and identifying gaps in understanding. It discusses the potential of RNA-based therapeutics to transform medicine, as well as the challenges associated with improving applicability, efficacy and safety profiles. Insights gained from RNA modalities such as antisense oligonucleotides (ASOs) and small interfering RNAs are used to identify important next steps for mRNA and gene editing technologies.

RNA 引发了现代医学的一场革命，有可能改变我们治疗疾病的方式。最近的监管批准、数百项新的临床试验、CRISPR 基因编辑的出现，以及 mRNA 疫苗在应对 COVID-19 大流行中的有效性，这些因素共同创造了巨大的动力和期望。然而，这类相对较新的药物的挑战仍然存在，需要专门的知识和专业知识来克服。本综述探讨了开发 RNA 药物平台的共同策略，包括分层技术、解决常见偏见和识别理解差距。它讨论了基于 RNA 的疗法改变医学的潜力，以及与提高适用性、有效性和安全性相关的挑战。从反义寡核苷酸 (ASO) 和小干扰 RNA 等 RNA 模式中获得的见解可用于确定 mRNA 和基因编辑技术的重要后续步骤。