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

• Main Authors: Da Sun (Author), Bhubanananda Sahu (Author), Songqi Gao (Author), Rebecca M. Schur (Author), Amita M. Vaidya (Author), Akiko Maeda (Author), Krzysztof Palczewski (Author), Zheng-Rong Lu (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: 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...