College of Pharmacy and Research Institute of Pharmaceutical Sciences, Seoul National University, Seoul, 08826, Republic of Korea.
College of Pharmacy, Jinan University, Guangzhou, 510632, China.
AAPS J. 2023 Oct 2;25(6):95. doi: 10.1208/s12248-023-00860-z.
Delivery of RNA using nanomaterials has emerged as a new modality to expand therapeutic applications in biomedical research. However, the delivery of RNA presents unique challenges due to its susceptibility to degradation and the requirement for efficient intracellular delivery. The integration of nanotechnologies with RNA delivery has addressed many of these challenges. In this review, we discuss different strategies employed in the design and development of nanomaterials for RNA delivery. We also highlight recent advances in the pharmaceutical applications of RNA delivered via nanomaterials. Various nanomaterials, such as lipids, polymers, peptides, nucleic acids, and inorganic nanomaterials, have been utilized for delivering functional RNAs, including messenger RNA (mRNA), small interfering RNA, single guide RNA, and microRNA. Furthermore, the utilization of nanomaterials has expanded the applications of functional RNA as active pharmaceutical ingredients. For instance, the delivery of antigen-encoding mRNA using nanomaterials enables the transient expression of vaccine antigens, leading to immunogenicity and prevention against infectious diseases. Additionally, nanomaterial-mediated RNA delivery has been investigated for engineering cells to express exogenous functional proteins. Nanomaterials have also been employed for co-delivering single guide RNA and mRNA to facilitate gene editing of genetic diseases. Apart from the progress made in RNA medicine, we discuss the current challenges and future directions in this field.
利用纳米材料传递 RNA 已经成为一种新的方式,可以扩展生物医学研究中的治疗应用。然而,由于 RNA 容易降解,并且需要有效的细胞内传递,因此传递 RNA 带来了独特的挑战。将纳米技术与 RNA 传递相结合,解决了许多这些挑战。在这篇综述中,我们讨论了设计和开发用于 RNA 传递的纳米材料时采用的不同策略。我们还强调了通过纳米材料传递的 RNA 在药物应用方面的最新进展。各种纳米材料,如脂质、聚合物、肽、核酸和无机纳米材料,已被用于传递功能性 RNA,包括信使 RNA(mRNA)、小干扰 RNA、单引导 RNA 和 microRNA。此外,纳米材料的利用扩展了功能性 RNA 作为活性药物成分的应用。例如,使用纳米材料传递抗原编码的 mRNA 可以使疫苗抗原瞬时表达,从而产生免疫原性并预防传染病。此外,纳米材料介导的 RNA 传递已被用于工程细胞表达外源性功能性蛋白。纳米材料还被用于共传递单引导 RNA 和 mRNA,以促进遗传疾病的基因编辑。除了在 RNA 医学方面取得的进展外,我们还讨论了该领域当前的挑战和未来方向。