脂质纳米颗粒的制备及在基因治疗中的应用

基因治疗已经成为人类治疗疾病的一种重要手段。然而，为了将基因药物用于临床，需要更加复杂的递送系统。脂质纳米颗粒(LNPs)系统是目前领先的非病毒递送系统，在治疗诊断学方面取得了许多令人鼓舞的进展，其具有实现基因药物临床治疗应用的潜力。由于LNPs纳米尺寸的优势及类脂化合物的生物相容性和生物降解性，LNPs能够克服阻碍基因药物应用中的障碍，安全而有效地递送核酸进入细胞，已经被广泛地应用于药物递送中。通过改进LNPs的制备方法、合理设计类脂化合物结构或优化LNPs配方将增加LNPs在基因治疗肿瘤以及其他疾病中的应用潜力。本文主要介绍LNPs的制备方法及在基因治疗中应用的最新进展。

Preparation of lipid nanoparticles and their application in gene therapy

Gene therapy has become an important method for humans to treat diseases. However, a complicated delivery system is required to use gene medicine in the clinic. The lipid nanoparticles (LNPs) system is currently the leading non-viral delivery system, and many encouraging advances have been made in therapeutic diagnostics, which can realize the clinical therapeutic potential of gene drugs. Due to the advantages of the size of the nanoparticles and the biocompatibility and biodegradability of the lipid, LNPs can overcome the obstacles that hinder the application of gene drugs to safely and effectively deliver nucleic acids into cells, and have been widely used in drug delivery. Improving the preparation method of LNPs, rationally designing the structure of lipid compounds, or optimizing the formulation of LNPs will increase the application potential of LNPs in gene therapy of tumors and other diseases. This article briefly introduces the preparation method of LNPs and the latest progress in the application of gene therapy.