Skin Cancer’s Personalized Topical Gene Therapy by Non-Viral Q-Starch/siRNA Nano-complexes

Nir Yarza
Chemical Engineering, Ben Gurion University, Israel

Skin cancers are currently treated mostly by infiltrative surgical methods, radiation and chemotherapy. Multiple tumor promoting pathways involved in melanoma and non-melanoma skin cancers, require invoking other strategies to detect prominent oncogenes of the diseases. RNA interference (RNAi) has the potential to serve as a therapeutic tool for treating skin cancer, but delivering functional RNAs to skin cells is challenging mainly due to the stratum corneum`s barrier properties. We therefore aim to exploit RNAi therapy, using ultrasound as a skin penetration enhancer, and modified polysaccharide/siRNA nano-complexes as a non-invasive, non-viral RNAi delivery system, that consequently downregulate specific oncogenes detected in melanoma and non-melanoma skin cancers, resulting in personalized gene therapy. Biocompatible and biodegradable potato starch was modified with positively charged quaternary amine groups (Q-starch) and electrostatically self-assembled with negatively charged siRNA to form complexes, that were characterized by gel electrophoresis, Zeta potential analyzer, DLS and FTIR. Q-starch/siRNA complexes were found to be ~80 nm in diameter with positive zeta potential, demonstrating nano-sized complexes capable of penetrating the cells` membrane, and indication of possible electrostatic interactions between the complexes and the negatively charged cell`s membrane. In vitro transfection and cellular uptake experiments on Basal Cell Carcinoma cell line (TE 354.T) and Melanoma cell line (a-375) measured by flow cytometry methods using fluorescently labeled complexes, demonstrated an efficient uptake of Q-starch/siRNACy5 complexes in various concentrations and their accumulation in the cytoplasm of the cells. RT-PCR demonstrated gene silencing in all concentrations, up to 70%, after 72 hours with Q-starch/siRNA nano-complexes.