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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Detalhes bibliográficos

Principais autores:	Da Sun, Bhubanananda Sahu, Songqi Gao, Rebecca M. Schur, Amita M. Vaidya, Akiko Maeda, Krzysztof Palczewski, Zheng-Rong Lu
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2017-06-01
coleção:	Molecular Therapy: Nucleic Acids
Assuntos:	Non-viral gene delivery multifunctional lipid gene replacement therapy Leber’s congenital amaurosis RPE65 protein
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2162253117301324
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