Journal
MOLECULAR PHARMACEUTICS
Volume 17, Issue 6, Pages 1848-1858Publisher
AMER CHEMICAL SOC
DOI: 10.1021/acs.molpharmaceut.9b01213
Keywords
nonviral vector; niosomes; VEGF; gene therapy; angiogenesis; central nervous system
Funding
- Basque Country Government (Department of Education, University and Research) [PRE_2016_2_0302, IT907-16]
- Research Chair Bidons Egara
- CIBER of Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN)
- Respiratory Diseases (CIBERES), an initiative of the Carlos III Health Institute (ISCIII)
- [RTI2018-098969-B-100]
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Gene therapy employing nanocarriers represents a promising strategy to treat central nervous system (CNS) diseases, where brain microvasculature is frequently compromised. Vascular endothelial growth factor (VEGF) is a key angiogenic molecule; however, its in vivo administration to the CNS by nonviral gene therapy has not been conducted. Hence, we prepared and physicochemically characterized four cationic niosome formulations (1-4), which were combined with pVEGF-GFP to explore their capacity to transfer the VEGF gene to CNS cells and achieve angiogenesis in the brain. Experiments in primary neuronal cells showed successful and safe transfection with niosome 4, producing double levels of biologically active VEGF in comparison to the rest of the formulations. Intracortical administration of niosome 4 based nioplexes in mouse brain validated the ability of this nonviral vector to deliver the VEGF gene to CNS cells, inducing brain angiogenesis and emerging as a promising therapeutic approach for the treatment of CNS diseases.
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