一种多功能抗体捕获系统,可实现 mRNA 脂质纳米颗粒的体内精确输送并增强基因表达能力

bioRxiv Pub Date : 2024-08-08 DOI:10.1101/2024.08.07.607101
M. Z. Chen, D. Yuen, Victoria M. McLeod, Ken W. Yong, Cameron H Smyth, Bruna Rossi Herling, Thomas Payne, Stewart A. Fabb, M. Belousoff, Azizah Algarni, Patrick M. Sexton, C. J. Porter, Colin W. Pouton, A. Johnston
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引用次数: 0

摘要

高效、精确地传递 mRNA 对于推动 mRNA 疗法超越其目前作为疫苗的用途至关重要。脂质纳米颗粒(LNP)能有效封装和保护 mRNA,但细胞的非特异性摄取可能会导致脱靶和对靶细胞的最小递送。用抗体对 LNP 进行功能化可实现 mRNA 的靶向递送,但传统的修饰技术需要复杂的共轭和纯化过程,往往会降低抗体的亲和力。在这里,我们提出了一种简单的方法,无需对抗体进行修饰或复杂的纯化,就能在 LNPs 上捕获最佳取向的抗体。这种策略在 LNP 表面使用最佳取向的抗 Fc 纳米抗体来捕获抗体,其蛋白质表达水平比非靶向 LNP 高出 1000 倍以上,比传统的抗体功能化技术高出 8 倍以上。这些精确靶向的 LNPs 在体内显示出对 T 细胞的高效靶向性,而对其他免疫细胞的传递则微乎其微。这种方法使靶向 LNPs 得以快速开发,并有可能扩大 mRNA 疗法的应用范围。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Versatile Antibody Capture System that Drives Precise In Vivo Delivery of mRNA loaded Lipid Nanoparticles and Enhances Gene Expression
Efficient and precise delivery of mRNA is critical to advance mRNA therapies beyond their current use as vaccines. Lipid nanoparticles (LNP) efficiently encapsulate and protect mRNA, but non-specific cellular uptake may lead to off-target delivery and minimal delivery to target cells. Functionalizing LNPs with antibodies enables targeted mRNA delivery, but traditional modification techniques require complex conjugation and purification, which often reduces antibody affinity. Here, we present a simple method for capturing antibodies in their optimal orientation on LNPs, without antibody modification or complex purification. This strategy uses an optimally oriented anti-Fc nanobody on the LNP surface to capture antibodies, resulting in protein expression levels >1000 times higher than non-targeted LNPs and >8 times higher than conventional antibody functionalization techniques. These precisely targeted LNPs showed highly efficient in vivo targeting to T cells, with minimal delivery to other immune cells. This approach enables the rapid development of targeted LNPs and has the potential to broaden the use of mRNA therapies.
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