Literature DB >> 26271011

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

Da Sun1, Hiroshi Maeno2, Maneesh Gujrati1, Rebecca Schur1, Akiko Maeda2,3, Tadao Maeda2, Krzysztof Palczewski3, Zheng-Rong Lu4.   

Abstract

Development of safe and effective gene delivery systems is essential in treating ocular genetic disorders. A hybrid nonpan>viral system composed of a multifunpan>ctionpan>al 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.
© 2015 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

Entities:  

Keywords:  DNA plasmid; ECO lipid; gene delivery; nanoglobule; subretinal gene delivery

Mesh：

Substances：

Year:  2015        PMID： 26271011      PMCID： PMC4770799          DOI： 10.1002/mabi.201500192

Source DB:  PubMed          Journal:  Macromol Biosci        ISSN： 1616-5187            Impact factor:   4.979


  37 in total

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2.  New amphiphilic carriers forming pH-sensitive nanoparticles for nucleic acid delivery.

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3.  Reduction of disulfide bonds within lysosomes is a key step in antigen processing.

Authors:  D S Collins; E R Unanue; C V Harding
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4.  Multiple internalization pathways of polyelectrolyte multilayer capsules into mammalian cells.

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Journal:  ACS Nano       Date:  2013-07-16       Impact factor: 15.881

5.  pH-triggered nanoparticle mediated delivery of siRNA to liver cells in vitro and in vivo.

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Journal:  Bioconjug Chem       Date:  2013-03-07       Impact factor: 4.774

6.  Synthesis and evaluation of a nanoglobular dendrimer 5-aminosalicylic Acid conjugate with a hydrolyzable schiff base spacer for treating retinal degeneration.

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Journal:  ACS Nano       Date:  2013-12-20       Impact factor: 15.881

Review 7.  Cationic lipids as transfecting agents of DNA in gene therapy.

Authors:  Elena Junquera; Emilio Aicart
Journal:  Curr Top Med Chem       Date:  2014-03       Impact factor: 3.295

Review 8.  Histones: structure and function.

Authors:  R J DeLange; E L Smith
Journal:  Annu Rev Biochem       Date:  1971       Impact factor: 23.643

9.  Copolymers of ethylene imine and N-(2-hydroxyethyl)-ethylene imine as tools to study effects of polymer structure on physicochemical and biological properties of DNA complexes.

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  17 in total

1.  Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles.

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2.  Stable Retinoid Analogue Targeted Dual pH-Sensitive Smart Lipid ECO/pDNA Nanoparticles for Specific Gene Delivery in the Retinal Pigment Epithelium.

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4.  Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber's Congenital Amaurosis.

Authors:  Da Sun; Bhubanananda Sahu; Songqi Gao; Rebecca M Schur; Amita M Vaidya; Akiko Maeda; Krzysztof Palczewski; Zheng-Rong Lu
Journal:  Mol Ther Nucleic Acids       Date:  2017-02-28

5.  Synthesis and Evaluation of pH-Sensitive Multifunctional Lipids for Efficient Delivery of CRISPR/Cas9 in Gene Editing.

Authors:  Da Sun; Zhanhu Sun; Hongfa Jiang; Amita M Vaidya; Rui Xin; Nadia R Ayat; Andrew L Schilb; Peter L Qiao; Zheng Han; Amirreza Naderi; Zheng-Rong Lu
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8.  Development of Sustained-Release Ophthalmic Formulation Based on Tranilast Solid Nanoparticles.

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10.  In Situ Gelling Systems Using Pluronic F127 Enhance Corneal Permeability of Indomethacin Nanocrystals.

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Journal:  Int J Mol Sci       Date:  2020-09-25       Impact factor: 5.923
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