期刊
MACROMOLECULAR BIOSCIENCE
卷 15, 期 12, 页码 1663-1672出版社
WILEY-V C H VERLAG GMBH
DOI: 10.1002/mabi.201500192
关键词
DNA plasmid; ECO lipid; gene delivery; nanoglobule; subretinal gene delivery
资金
- National Eye Institute of the National Institutes of Health (NIH) [R24EY021126]
Development of safe and effective gene delivery systems is essential in treating ocular genetic disorders. A hybrid nonviral system composed of a multifunctional lipid ECO and a G4 nanoglobule was designed for efficient gene delivery into RPE cells at low charge ratios. This system formed stable DNA nanoparticles at low N/P ratios, exhibited low cytotoxicity, and induced higher GFP expression in ARPE-19 cells at N/P = 6. The hybrid nanoparticles mediated significant reporter gene GFP expression ex-vivo in the retina from wild type C57 mice and in vivo in BALB/c mice. These hybrid nanoparticles are promising for in vitro and in vivo gene delivery at low charge ratios.
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