Practical Clinical Approaches Used in RNA-Based Therapeutics
摘要
In a variety of disorders, RNAs offer significant potential as therapeutic agents to broaden the scope of traditional therapeutic targets, ranging from external and surface proteins to intracellular nucleic acids and their regulators. The adaptability of RNA can be used to identify different cell types, carry out cell treatment, and create novel vaccine classes. Antisense nucleotides, aptamers, miRNA, mRNA, and siRNA are examples of therapeutic RNAs that can alter or trigger protein expression, prevent molecular interactions, accomplish genome editing, and bypass exons. A common RNA thread is its structure, flexibility, and binding selectivity, which make it extremely attractive for therapeutic applications. Furthermore, RNA exhibits unique structural flexibility in contrast to both DNA and proteins. The diagnosis and treatment of human disorders could be revolutionized by RNA-based medicines. Even though early research had difficulties, it laid the groundwork for advances in RNA-based drug design, which led to the incredibly quick creation of mRNA vaccines to fight the COVID-19 pandemic. At this critical juncture, RNA medications have the potential to have a significant clinical impact. This chapter provides a thorough description of the many kinds of RNA-based treatments. We also provide some information on their production, use, impacts, and drawbacks.