Lundstrom Kenneth
PanTherapeutics, Rue des Remparts 4, CH1095 Lutry, Switzerland.
Expert Rev Vaccines. 2015 Feb;14(2):253-63. doi: 10.1586/14760584.2015.959932. Epub 2014 Sep 14.
RNA-based approaches have provided novel alternatives for modern drug discovery. The application of RNA as therapeutic agents has, until recently, been hampered by issues related to poor delivery and stability, but chemical modifications and new delivery approaches have increased progress. Moreover, the discovery of the importance of RNA in gene regulation and gene silencing has revealed new drug targets, especially related to treatment of cancer and other diseases. Recent engineering of small molecules designed from RNA sequences to target miRNAs opens up new possibilities in drug development. Furthermore, RNA-based vaccines have been engineered applying RNA virus vectors and non-viral delivery for vaccine development.
基于RNA的方法为现代药物发现提供了新的选择。直到最近,RNA作为治疗剂的应用一直受到递送和稳定性差等问题的阻碍,但化学修饰和新的递送方法已取得了进展。此外,RNA在基因调控和基因沉默中的重要性的发现揭示了新的药物靶点,特别是与癌症和其他疾病的治疗相关的靶点。最近从RNA序列设计小分子以靶向miRNA的工程化开辟了药物开发的新可能性。此外,基于RNA的疫苗已通过应用RNA病毒载体和非病毒递送进行疫苗开发。