Niazi Sarfaraz K, Magoola Matthias
College of Pharmacy, University of Illinois, Chicago, IL 60612, USA.
DEI Biopharma, Kampala P.O. Box 35854, Uganda.
Int J Mol Sci. 2024 Nov 29;25(23):12883. doi: 10.3390/ijms252312883.
MicroRNAs (miRNAs) maintain cellular homeostasis by blocking mRNAs by binding with them to fine-tune the expression of genes across numerous biological pathways. The 2024 Nobel Prize in Medicine and Physiology for discovering miRNAs was long overdue. We anticipate a deluge of research work involving miRNAs to repeat the history of prizes awarded for research on other RNAs. Although miRNA therapies are included for several complex diseases, the realization that miRNAs regulate genes and their roles in addressing therapies for hundreds of diseases are expected; but with advancement in drug discovery tools, we anticipate even faster entry of new drugs. To promote this, we provide details of the current science, logic, intellectual property, formulations, and regulatory process with anticipation that many more researchers will introduce novel therapies based on the discussion and advice provided in this paper.
微小RNA（miRNA）通过与信使核糖核酸（mRNA）结合来阻断它们，从而维持细胞内稳态，以微调众多生物途径中基因的表达。因发现miRNA而授予的2024年诺贝尔生理学或医学奖早就该颁发了。我们预计会有大量涉及miRNA的研究工作，重演因其他RNA研究而获奖的历史。尽管miRNA疗法已被应用于几种复杂疾病，但预计miRNA对基因的调控及其在数百种疾病治疗中的作用将会得到进一步认识；不过随着药物研发工具的进步，我们预计新药的上市速度会更快。为推动这一进程，我们提供了当前科学、逻辑、知识产权、制剂和监管流程的详细信息，期望更多研究人员能基于本文的讨论和建议推出新的疗法。
