Lipopolyplex for Therapeutic Gene Delivery and Its Application for the Treatment of Parkinson's Disease.
Lipopolyplex is a core-shell structure composed of nucleic acid, polycation and lipid. As a non-viral gene delivery vector, lipopolyplex combining the advantages of polyplex and lipoplex has shown superior colloidal stability, reduced cytotoxicity, extremely high gene transfection efficiency. Following intravenous administration, there are many strategies based on lipopolyplex to overcome the complex biological barriers in systemic gene delivery including condensation of nucleic acids into nanoparticles, long circulation, cell targeting, endosomal escape, release to cytoplasm and entry into cell nucleus. Parkinson's disease (PD) is the second most common neurodegenerative disorder and severely influences the patients' life quality. Current gene therapy clinical trials for PD employing viral vectors didn't achieve satisfactory efficacy. However, lipopolyplex may become a promising alternative approach owing to its stability in blood, ability to cross the blood-brain barrier (BBB) and specific targeting to diseased brain cells.
Department of Neurology, Xinhua Hospital, Shanghai JiaoTong University School of Medicine Shanghai, China.
Wei
W
Chen
School of Pharmacy, Shanghai JiaoTong University Shanghai, China.
Hui
H
Li
Department of Neurology, Xinhua Hospital, Shanghai JiaoTong University School of Medicine Shanghai, China.
Zhenguo
Z
Liu
School of Pharmacy, Shanghai JiaoTong University Shanghai, China.
Weien
W
Yuan
Parkinson’s disease; biological barriers; blood brain barrier; lipopolyplex