Niazi Sarfaraz K
College of Pharmacy, University of Illinois, Chicago, IL 60612, USA.
Biomedicines. 2023 Apr 25;11(5):1275. doi: 10.3390/biomedicines11051275.
COVID-19 brought about the mRNA vaccine and a paradigm shift to a new mode of treating and preventing diseases. Synthetic RNA products are a low-cost solution based on a novel method of using nucleosides to act as an innate medicine factory with unlimited therapeutic possibilities. In addition to the common perception of vaccines preventing infections, the newer applications of RNA therapies include preventing autoimmune disorders, such as diabetes, Parkinson's disease, Alzheimer's disease, and Down syndrome; now, we can deliver monoclonal antibodies, hormones, cytokines, and other complex proteins, reducing the manufacturing hurdles associated with these products. Newer PCR technology removes the need for the bacterial expression of DNA, making mRNA a truly synthetic product. AI-driven product design expands the applications of mRNA technology to repurpose therapeutic proteins and test their safety and efficacy quickly. As the industry focuses on mRNA, many novel opportunities will arise, as hundreds of products under development will bring new perspectives based on this significant paradigm shift-finding newer solutions to existing challenges in healthcare.
新冠疫情催生了信使核糖核酸（mRNA）疫苗，并引发了治疗和预防疾病新模式的范式转变。合成RNA产品是一种低成本解决方案，基于一种利用核苷作为天然药厂的新方法，具有无限的治疗可能性。除了疫苗预防感染的普遍认知外，RNA疗法的新应用还包括预防自身免疫性疾病，如糖尿病、帕金森病、阿尔茨海默病和唐氏综合征；现在，我们可以递送单克隆抗体、激素、细胞因子和其他复杂蛋白质，减少与这些产品相关的制造障碍。更新的聚合酶链式反应（PCR）技术不再需要细菌表达DNA，使mRNA成为真正的合成产品。人工智能驱动的产品设计扩展了mRNA技术的应用，以重新利用治疗性蛋白质并快速测试其安全性和有效性。随着该行业专注于mRNA，许多新机遇将会出现，因为数百种正在研发的产品将基于这一重大范式转变带来新的视角——为医疗保健领域现有的挑战找到更新的解决方案。
