Department of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
Department of Medico-Surgical Sciences and Biotechnology, Sapienza University of Rome, Laboratory affiliated to Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Latina, Italy.
Nat Commun. 2024 Oct 23;15(1):9119. doi: 10.1038/s41467-024-53569-8.
Lipid nanoparticles (LNPs) play a crucial role in addressing genetic disorders, and cancer, and combating pandemics such as COVID-19 and its variants. Yet, the ability of LNPs to effectively encapsulate large-size DNA molecules remains elusive. This is a significant limitation, as the successful delivery of large-size DNA holds immense potential for gene therapy. To address this gap, the present study focuses on the design of PEGylated LNPs, incorporating large-sized DNA, departing from traditional RNA and ionizable lipids. The resultant LNPs demonstrate a unique particle morphology. These particles were further engineered with a DNA coating and plasma proteins. This multicomponent bionanoconstruct exhibits enhanced transfection efficiency and safety in controlled laboratory settings and improved immune system evasion in in vivo tests. These findings provide valuable insights for the design and development of bionanoarchitectures for large-size DNA delivery, opening new avenues for transformative gene therapies.
脂质纳米颗粒 (LNPs) 在解决遗传疾病、癌症以及应对 COVID-19 及其变体等大流行方面发挥着关键作用。然而,LNPs 有效包裹大尺寸 DNA 分子的能力仍然难以捉摸。这是一个重大的限制,因为成功输送大尺寸 DNA 为基因治疗提供了巨大的潜力。为了解决这一差距,本研究专注于设计聚乙二醇化 LNPs,将大尺寸 DNA 纳入其中,而不是传统的 RNA 和可离子化脂质。所得的 LNPs 表现出独特的颗粒形态。这些颗粒进一步用 DNA 涂层和血浆蛋白进行工程化处理。这种多组分仿生纳米结构在受控的实验室环境中表现出增强的转染效率和安全性,并在体内试验中提高了免疫系统逃避能力。这些发现为大尺寸 DNA 传递的仿生纳米结构的设计和开发提供了有价值的见解,为变革性基因治疗开辟了新途径。