Self-Assembly of a Multifunctional Lipid With Core-Shell Dendrimer DNA Nanoparticles Enhanced Efficient Gene Delivery at Low Charge Ratios into RPE Cells

Autor:	Hiroshi Maeno, Maneesh Gujrati, Krzysztof Palczewski, Tadao Maeda, Akiko Maeda, Zheng-Rong Lu, Da Sun, Rebecca M. Schur
Rok vydání:	2015
Předmět:	Reporter gene Polymers and Plastics Wild type Bioengineering Nanotechnology Gene delivery Biology In vitro Green fluorescent protein Biomaterials In vivo Dendrimer Materials Chemistry Biophysics Cytotoxicity Biotechnology
Zdroj:	Macromolecular Bioscience. 15:1663-1672
ISSN:	1616-5187
DOI:	10.1002/mabi.201500192
Popis:	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.
Databáze:	OpenAIRE
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