Glucocorticoid pre-administration improves LNP-mRNA mediated protein replacement and genome editing therapies.

 0 Người đánh giá. Xếp hạng trung bình 0

Tác giả: Xiaobo Cen, Changyang Gong, Yaoge Jiao, Li Li, Mei Luo, Chunting Wang, Yanhong Wang, Shaohua Yao, Lifang Zhou

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: Netherlands : International journal of pharmaceutics , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 730950

Lipid nanoparticles (LNPs) are among the most promising non-viral mRNA delivery systems for gene therapeutic applications. However, the in vivo delivery of LNP-mRNA remains challenging due to multiple intrinsic barriers that hinder LNPs from reaching their target cells. In this study, we sought to enhance LNP delivery by manipulating intrinsic regulatory mechanisms involved in their metabolism. We demonstrated that activation of the glucocorticoid pathway significantly increased the systemic delivery of LNP-mRNA in both mice and monkeys, achieving up to a fourfold improvement. This enhancement was primarily attributed to the glucocorticoid-mediated inhibition of macrophage phagocytosis in circulation and the liver, which resulted in higher LNP accumulation in hepatocytes. Consequently, glucocorticoid activation improved the therapeutic efficacy of LNP-based protein replacement and CRISPR/Cas9 genome editing therapies. Together, these findings establish a practical strategy to enhance the systemic delivery of RNA-based protein replacement and genome editing therapeutics, highlighting the potential of manipulating endogenous mechanisms to optimize exogenous gene delivery.
Tạo bộ sưu tập với mã QR

THƯ VIỆN - TRƯỜNG ĐẠI HỌC CÔNG NGHỆ TP.HCM

ĐT: (028) 36225755 | Email: tt.thuvien@hutech.edu.vn

Copyright @2024 THƯ VIỆN HUTECH