Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing

• Tác giả: Mohamad-Gabriel Alameh, Vivek Chowdhary, Ningqiang Gong, Xuexiang Han, Michael J Mitchell, Rohan Palanki, William H Peranteau, Adele Ricciardi, Drew Weissman, James M Wilson, Junchao Xu, Ying Xu, Lulu Xue
• Ngôn ngữ: eng
• Ký hiệu phân loại: 070.51 Selection and editing of manuscripts
• Thông tin xuất bản: United States : bioRxiv : the preprint server for biology , 2025
• Bộ sưu tập: NCBI
• ID: 683571

Tóm tắt

mRNA-based gene editing therapeutics offer the potential to permanently cure diseases but are hindered by suboptimal delivery platforms. Here, we devise a robust combinatorial chemistry for plug-and-play assembly of diverse biodegradable ionizable lipids and identify a lead candidate that produces superior lipid nanoparticles for various gene editing tools delivery