Defensins are small cysteine-rich innate immune peptides with antimicrobial and anticancer activity. Plant defensins such as NaD1 [1] and TPP3 [2], as well as human β-defensins HBD2 [3] and HBD3 [4], have been shown to exert their activity by interacting with specific anionic membrane phospholipids, known as phosphoinositides. Phosphoinositides are minor membrane lipids that are crucial for growth, proliferation and survival of the cell and are often dysregulated in microbes and tumour cells [5, 6], and may be targeted for therapeutic application. In this study, human β-defensins HBD9 and HBD14 have been characterised by determining their phosphoinositide-binding specificity and cytotoxic activity against fungal and tumour cells.
HBD9 and HBD14 were successfully expressed and purified using pHUE vector in BL21 CodonPlus (RIL) cells. The quality, purity and integrity of the defensins were confirmed by SDS-PAGE, immunoblot and mass spectrometry analyses. Protein-lipid overlay assay revealed that HBD9 binds specifically to phosphatidylinositol-4-phosphate (PI4P), whereas HBD14 bound to a broad range of different phospholipids, including phosphatidic acid (PA) and phosphatidylinositol-4,5-bisphoaphate (PI(4,5)P₂). HBD9 was shown to be active against human tumour cell lines (PC3, HeLa, MDA-MB-231 and MCF-7) and the human fungal pathogen Candida albicans strains (ATCC10231 and ATCC90028) at low micromolar concentrations (0.3-1.3 μM). HBD14 was also active against the tumour cell lines and C. albicans but at higher micromolar concentrations (15-72 μM). Membrane permeabilisation assay showed uptake of the membrane impermeable dye, propidium iodide (PI) in MDA-MB-231 and HeLa cells treated with HBD9 and HBD14, demonstrating that these defensins act by destabilising membranes.
The data presented in this study provides proof of concept that defensins have a conserved mode of action, where they exert their activity by binding to membrane phospholipids, causing membrane disruption and cell death.