Multi-stage-mixing to control the supramolecular structure of lipid nanoparticles, thereby creating a core-then-shell arrangement that improves performance by orders of magnitude.

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Tác giả: Elena N Atochina-Vasserman, Jacob S Brenner, Quang Minh Dang, Xijing Gong, Kushol Gupta, Andrew Hanna, Hui-Ting Huang, David Issadore, Oscar A Marcos-Contreras, Jonathan J Miner, Kahlilia C Morris-Blanco, Vladimir R Muzykantov, Jacob W Myerson, Jia Nong, Wook-Jin Park, Manthan Patel, Sachchidanand Tiwari, Zhicheng Wang, Drew Weissman, Jichuan Wu

Ngôn ngữ: eng

Ký hiệu phân loại: 322.5 Armed services

Thông tin xuất bản: United States : bioRxiv : the preprint server for biology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 159708

As they became the dominant gene therapy platform, lipid nanoparticles (LNPs) experienced nearly all their innovation in varying the structure of individual molecules in LNPs. This ignored control of the spatial arrangement of molecules, which is suboptimal because supramolecular structure determines function in biology. To control LNPs' supramolecular structure, we introduce multi-stage-mixing (MSM) to successively add different molecules to LNPs. We first utilize MSM to create a core-then-shell (CTS) synthesis. CTS-LNPs display a clear core-shell structure, vastly lower frequency of LNPs containing no detectable mRNA, and improved mRNA-LNP expression. With DNA-loaded LNPs, which for decades lagged behind mRNA-LNPs due to low expression, CTS improved DNA-LNPs' protein expression by 2-3 orders of magnitude, bringing it within range of mRNA-LNPs. These results show that supramolecular arrangement is critical to LNP performance and can be controlled by mixing methodology. Further, MSM/CTS have finally made DNA-LNPs into a practical platform for long-term gene expression.
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