An MRI-Visible Non-Viral Vector Bearing GD2 Single Chain Antibody for Targeted Gene Delivery to Human Bone Marrow Mesenchymal Stem Cells

作者：Pang, Pengfei; Wu, Chun; Shen, Min; Gong, Faming; Zhu, Kangshun; Jiang, Zaibo; Guan, Shouhai; Shan, Hong^*; Shuai, Xintao

来源：PLos One, 2013, 8(10): e76612.

DOI：10.1371/journal.pone.0076612

• NEURAL GANGLIOSIDE GD2
• STROMAL CELLS
• UMBILICAL-CORD
• DNA
• CYTOTOXICITY
• EXPRESS
• DISEASE
• CULTURE
• TRAIL
• PEI

The neural ganglioside GD2 has recently been reported to be a novel surface marker that is only expressed on human bone marrow mesenchymal stem cells within normal marrow. In this study, an MRI-visible, targeted, non-viral vector for effective gene delivery to human bone marrow mesenchymal stem cells was first synthesized by attaching a targeting ligand, the GD2 single chain antibody (scAb(GD2)), to the distal ends of PEG-g-PEI-SPION. The targeted vector was then used to condense plasmid DNA to form nanoparticles showing stable small size, low cytotoxicity, and good biocompatibility. Based on a reporter gene assay, the transfection efficiency of targeting complex reached the highest value at 59.6% +/- 4.5% in human bone marrow mesenchymal stem cells, which was higher than those obtained using nontargeting complex and lipofectamine/pDNA (17.7% +/- 2.9% and 34.9% +/- 3.6%, respectively) (P<0.01). Consequently, compared with the nontargeting group, more in vivo gene expression was observed in the fibrotic rat livers of the targeting group. Furthermore, the targeting capacity of scAb(GD2)-PEG-g-PEI-SPION was successfully verified in vitro by confocal laser scanning microscopy, Prussian blue staining, and magnetic resonance imaging. Our results indicate that scAb(GD2)-PEG-g-PEI-SPION is a promising MRI-visible non-viral vector for targeted gene delivery to human bone marrow mesenchymal stem cells.

• 出版日期2013-10-7
• 单位中山大学