Tsakiri Maria, Zivko Cristina, Demetzos Costas, Mahairaki Vasiliki
Section of Pharmaceutical Technology, Department of Pharmacy, School of Health Sciences, National and Kapodistrian University of Athens, Athens, Greece.
Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, United States.
Front Pharmacol. 2022 Aug 9;13:900610. doi: 10.3389/fphar.2022.900610. eCollection 2022.
RNA-delivery is a promising tool to develop therapies for difficult to treat diseases such as neurological disorders, by silencing pathological genes or expressing therapeutic proteins. However, in many cases RNA delivery requires a vesicle that could effectively protect the molecule from bio-degradation, bypass barriers i.e., the blood brain barrier, transfer it to a targeted tissue and efficiently release the RNA inside the cells. Many vesicles such as viral vectors, and polymeric nanoparticles have been mentioned in literature. In this review, we focus in the discussion of lipid-based advanced RNA-delivery platforms. Liposomes and lipoplexes, solid lipid nanoparticles and lipid nanoparticles are the main categories of lipidic platforms for RNA-delivery to the central nervous systems (CNS). A variety of surface particles' modifications and routes of administration have been studied to target CNS providing encouraging results . It is concluded that lipid-based nanoplatforms will play a key role in the development of RNA neuro-therapies.
RNA递送是一种很有前景的工具，可通过沉默致病基因或表达治疗性蛋白质来开发针对神经系统疾病等难治性疾病的疗法。然而，在许多情况下，RNA递送需要一种囊泡，它能够有效地保护分子不被生物降解，绕过诸如血脑屏障等屏障，将其转移到靶向组织，并在细胞内有效释放RNA。文献中提到了许多囊泡，如病毒载体和聚合物纳米颗粒。在本综述中，我们重点讨论基于脂质的先进RNA递送平台。脂质体和脂质复合物、固体脂质纳米颗粒和脂质纳米颗粒是用于向中枢神经系统（CNS）递送RNA的脂质平台的主要类别。为了靶向中枢神经系统，已经研究了各种表面颗粒修饰和给药途径，并取得了令人鼓舞的结果。结论是，基于脂质的纳米平台将在RNA神经疗法的开发中发挥关键作用。
