CD47 Peptide-Cloaked Lipid Nanoparticles Promote Cell-Specific mRNA Delivery.

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Tác giả: Awurama Akyianu, Laura Breda, Qin Li, Barbara L Mui, Vladimir R Muzykantov, Tyler Ellis Papp, Hamideh Parhiz, Stefano Rivella, Hamna Shahnawaz, Ruiqi Shi, Vladimir Shuvaev, Joseph Steward, Ying K Tam, Drew Weissman, Amir Yadegari, Jianhao Zeng

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : Molecular therapy : the journal of the American Society of Gene Therapy , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 712788

mRNA-based therapeutics delivered via lipid nanoparticles (LNP-mRNA) hold great promise for treating diverse diseases. However, further improvements are needed to refine outcomes in non-vaccine, extrahepatic applications, such as minimizing the mononuclear phagocyte system's (MPS)' rapid clearance and off-target toxicity in undesired tissues. We propose modifying LNP surfaces with the phagocytic cell "don't eat me" signal, CD47, in combination with our previously established antibody-based targeted LNP (tLNP) to create a CD47/tLNP platform with reduced phagocytic clearance and off-target effects, and improved efficiency for cell-specific delivery. We showed that CD47 modification decreased macrophage and hepatic uptake both in vitro and in vivo. Combining CD47 modification with antibodies targeting endothelial cells, T cells, or hematopoietic stem cells (HSCs) increased targeting efficiency up to 3-fold compared to tLNP alone. Enhanced targeting of CD47/tLNP to HSCs with reduced off-targeting enabled the delivery of pro-apoptotic mRNA for HSC depletion as a preconditioning strategy prior to bone marrow transplant. Additionally, CD47-modified LNPs showed diminished inflammatory effects on hepatic tissue and an altered protein corona. Our CD47/tLNP-mRNA platform, with its reduced phagocytic clearance, mitigated inflammatory effects, and enhanced targeted delivery, should further facilitate the development of in vivo mRNA therapeutics.
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