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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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Detalles Bibliográficos
Autores principales:	Sun, Da, Sahu, Bhubanananda, Gao, Songqi, Schur, Rebecca M., Vaidya, Amita M., Maeda, Akiko, Palczewski, Krzysztof, Lu, Zheng-Rong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363681/ https://www.ncbi.nlm.nih.gov/pubmed/28624218 http://dx.doi.org/10.1016/j.omtn.2017.02.005

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