Liver cancer is one of the most common
malignancies worldwide. The
RAF kinase inhibitors are effective in the treatment of
hepatocellular carcinoma (HCC); therefore, inhibition of the BRAF/MEK/ERK pathway has become a new therapeutic strategy for novel HCC
therapy. However, targeted specific delivery systems for
tumors are still significant obstacle to clinical applications.
Galactose (GAL) can target the
asialoglycoprotein receptor (ASGPR) that is highly expressed on
liver cancer cells. In this study, we designed a novel multifunctional nanomaterial GAL-GNR-siBRAF which consists of three parts, GAL as the
liver cancer-targeting moiety, golden nanorods (GNR) offering photothermal capability under near infrared light, and
siRNA specifically silencing BRAF (siBRAF). The nanocarrier GAL-GNR-siBRAF showed high
siRNA loading capacity and inhibited the degradation of
siRNA in serum. Compared with naked
gold nanorods, GAL-GNR-siBRAF possessed lower biotoxicity and higher efficacy of gene silencing. Treatment with GAL-GNR-siBRAF significantly downregulated the expression of BRAF and impaired proliferation, migration, and invasion of
liver cancer cells. Moreover, combinatorial photothermal effects and BRAF knockdown by GAL-GNR-siBRAF effectively given rise to
tumor cell death. Therefore, our study developed a new type of targeted multi-functional nanomaterial GAL-GNR-siBRAF for the treatment of
liver cancer, which provides ideas for the development of new clinical treatment methods.