DQAsomes as a coenzyme Q10 delivery vehicle: A step forward therapy in leigh disease

Abstract

Leigh syndrome (LS), a severe neurometabolic disorder caused by mitochondrial dysfunction, is often linked to coenzyme Q10 (CoQ10) defciency. This study investigated using dequalinium chloride to create CoQ10-loaded DQAsomes (liposome like vesicles) to improve CoQ10 solubility and delivery to mitochondria. DQAsomes were characterized for size, charge, encapsulation efciency, and yield. Further, drug release, dissolution, and efects on cell viability were studied in human pluripotent stem cells (HPP) and NDUFV gene-mutant cells (MDCi007-A). Techniques like RT-PCR, ELISA, immu nostaining, and Western blotting were used to assess pluripotency markers, gene expression, and apoptosis. Results showed DQAsomes with sizes between 165.8 and 311.2 nm and a negative charge. Encapsulation efciencies ranged from 34.03 to 82.48%. CoQ10-loaded DQAsomes signifcantly improved cell viability compared to CoQ10 in solution. Additionally, CoQ10-DQAsomes decreased pluripotency markers, suggesting potential efects on stem cell properties. In summary, the development of CoQ10-loaded DQAsomes ofers a promising approach to enhance CoQ10 delivery, potentially improving cellular health in LS. This represents a signifcant step forward in exploring new treatments for this challenging condition.