Multifunctional gene delivery vectors containing different liver-targeting fragments for specifically transfecting hepatocellular carcinoma (HCC) cells

Gene therapy is a promising strategy for HCC treatment, but it commonly faces the problem of low specificity in gene transfection. In this study, we designed and synthesized a series of peptide-based gene delivery vectors (H-01 to H-18) containing varied HCC cell-targeting fragments for specifically binding different receptors highly expressed on HCC cell membranes. The physicochemical properties of peptide vectors or peptide/DNA complexes were characterized, and the gene delivery abilities of peptide vectors were evaluated in HepG2 cell lines. The results showed that peptide vectors H-02 and H-09, which contained targeted fragments for ACE2 and c-Met receptors, respectively, could specifically transfect HCC cells in a highly -efficient manner in vitro. Furthermore, the liver-targeting function in vivo of Cy5.5 labeled H-02 (H-17) and H-09 (H-18) was investigated by fluorescence imaging. Multifunctional peptide-based materials achieved targeted gene delivery into hepatocellular carcinoma (HCC) cells efficiently.

Automated summary

Gene therapy is a promising strategy for treating HCC, but it often lacks specificity in gene transfection. In this study, a series of peptide-based gene delivery vectors were designed and synthesized to specifically bind receptors highly expressed on HCC cell membranes. The results showed that peptide vectors containing targeted fragments for ACE2 and c-Met receptors were able to efficiently transfect HCC cells in vitro. Furthermore, the liver-targeting function of these peptides was evaluated in vivo using fluorescence imaging.