Da Sun1, Hiroshi Maeno2, Maneesh Gujrati1, Rebecca Schur1, Akiko Maeda2,3, Tadao Maeda2, Krzysztof Palczewski3, Zheng-Rong Lu4. 1. Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio , 44140, USA. 2. Department of Ophthalmology, School of Medicine, Case Western Reserve University, Cleveland, Ohio, 44140, USA. 3. Department of Pharmacology, Cleveland Center for Membrane and Structural Biology, School of Medicine, Case Western Reserve University, Cleveland, Ohio, 44140, USA. 4. Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, Ohio , 44140, USA. zxl125@case.edu.
Abstract
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.
Development of safe and effective gene delivery systems is essential in treating n class="Disease">ocular genetic disorders. A hybrid nonpan>viral system composed of a multifunpan>ctionpan>al pan> class="Chemical">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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