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

• Päätekijät: Da Sun (Tekijä), Bhubanananda Sahu (Tekijä), Songqi Gao (Tekijä), Rebecca M. Schur (Tekijä), Amita M. Vaidya (Tekijä), Akiko Maeda (Tekijä), Krzysztof Palczewski (Tekijä), Zheng-Rong Lu (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: Elsevier, 2017-06-01T00:00:00Z.
• Aiheet: article
• Linkit: Connect to this object online.

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 mo...