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
Publicado no:Mol Ther Nucleic Acids
Main Authors: Sun, Da, Sahu, Bhubanananda, Gao, Songqi, Schur, Rebecca M., Vaidya, Amita M., Maeda, Akiko, Palczewski, Krzysztof, Lu, Zheng-Rong
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363681/
https://ncbi.nlm.nih.gov/pubmed/28624218
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.02.005
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