대한신장학회

논문분류	춘계학술대회 초록집
제목	Hybrid Lipid Nanoparticle-Based Delivery System for Targeted CRISPR/Cas9 Gene Therapy in Kidney Endothelial Cells
저자	Jee-Yeon Ryu
출판정보	2025; 2025(1):
키워드	lipid nanoparticle, CRISPR/Cas9 , gene therapy, kidney endothelial cells
초록	Gene therapy using the CRISPR/Cas9 system holds great promise for treating various diseases, but practical limitations remain, especially regarding effective delivery systems. The kidney presents challenges for targeted therapies due to its structural complexity, specialized cell types, and selective filtration barriers. To address these issues, we developed a lipid nanoparticle (LNP)-based delivery system hybridized with red blood cell (RBC) membranes for diabetic kidney disease (DKD) treatment. To improve kidney-specific targeting, we developed an LNP-based delivery system hybridized with RBC membranes and conjugated the LNPs with vascular endothelial protein tyrosine phosphatase (VE-PTP) antibodies, enabling selective delivery to kidney endothelial cells. Furthermore, the LNPs were designed to encapsulate and deliver the CRISPR/Cas9 system to target angiopoietin-1 (ANGPT1) for gene therapy to restore vascular homeostasis and enhance kidney function. These nanoparticles were tested on human kidney endothelial cells to assess their potential for gene therapy applications. Human kidney endothelial cells efficiently internalized the hybrid LNP-RBC membrane system in vitro, demonstrating its effectiveness in facilitating cellular uptake. Moreover, the LNPs successfully delivered the gene-editing tool to endothelial cells. This approach effectively induced ANGPT1 up-regulation and regulated downstream signaling pathways. The system also modulated cell viability and migration using WST-1 assay and migration assay. These findings highlight the potential of the LNP platform to deliver gene therapies effectively and regulate key biological processes in kidney endothelial cells. This study demonstrates the potential of a hybrid LNP-based delivery system as a novel and effective platform for gene therapy, particularly for delivering gene-editing tools such as the CRISPR/Cas9 system. By hybridizing LNPs with RBC membranes, we enhanced nanoparticle stability and targeted delivery to endothelial cells. Further research is required to evaluate the long-term safety, efficacy, and broader applications of this innovative delivery system in vivo.
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