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

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

Development of a gene delivery system with high efficiency and a good safety profile is essential for successful gene therapy. Here we developed a targeted non-viral delivery system using a multifunctional lipid ECO for treating Leber’s congenital amaurosis type 2 (LCA2) and tested this in a mouse m...

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Bibliographic Details
Main Authors: Da Sun, Bhubanananda Sahu, Songqi Gao, Rebecca M. Schur, Amita M. Vaidya, Akiko Maeda, Krzysztof Palczewski, Zheng-Rong Lu
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
Non-viral gene delivery
multifunctional lipid
gene replacement therapy
Leber’s congenital amaurosis
RPE65 protein
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301324

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