Department of Pharmacy, Aspetar Orthopedic and Sports Medicine Hospital, Doha, Qatar.
Department of Pharmaceutical Sciences, College of Pharmacy, QU Health, Qatar University, Doha, Qatar.
Biomed Pharmacother. 2022 Jan;145:112385. doi: 10.1016/j.biopha.2021.112385. Epub 2021 Oct 28.
Chemically modified mRNA represents a unique, efficient, and straightforward approach to produce a class of biopharmaceutical agents. It has been already approved as a vaccination-based method for targeting SARS-CoV-2 virus. The COVID-19 pandemic has highlighted the prospect of synthetic modified mRNA to efficiently and safely combat various diseases. Recently, various optimization advances have been adopted to overcome the limitations associated with conventional gene therapeutics leading to wide-ranging applications in different disease conditions. This review sheds light on emerging directions of chemically modified mRNAs to prevent and treat widespread chronic diseases, including metabolic disorders, cancer vaccination and immunotherapy, musculoskeletal disorders, respiratory conditions, cardiovascular diseases, and liver diseases.
化学修饰的 mRNA 代表了一种独特、高效和直接的方法来生产一类生物制药制剂。它已经被批准为针对 SARS-CoV-2 病毒的基于疫苗的方法。COVID-19 大流行突出了合成修饰 mRNA 的前景，可以有效地、安全地治疗各种疾病。最近，各种优化进展被采用，以克服与传统基因治疗相关的限制，从而在不同疾病情况下得到广泛应用。本文综述了化学修饰的 mRNA 在预防和治疗广泛的慢性疾病方面的新兴方向，包括代谢紊乱、癌症疫苗接种和免疫疗法、肌肉骨骼疾病、呼吸状况、心血管疾病和肝脏疾病。
