An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.
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ABSTRACT:
SUBMITTER: Li B
PROVIDER: S-EPMC4869688 | biostudies-literature | 2015 Dec
REPOSITORIES: biostudies-literature
Publications
An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.
Li Bin B Li Bin B Luo Xiao X Deng Binbin B Wang Junfeng J McComb David W DW Shi Yimin Y Gaensler Karin M L KM Tan Xu X Dunn Amy L AL Kerlin Bryce A BA Dong Yizhou Y
Nano letters 20151106 12
Systemic delivery of mRNA-based therapeutics remains a challenging issue for preclinical and clinical studies. Here, we describe new lipid-like nanoparticles (TT-LLNs) developed through an orthogonal array design, which demonstrates improved delivery efficiency of mRNA encoding luciferase in vitro by over 350-fold with significantly reduced experimental workload. One optimized TT3 LLN, termed O-TT3 LLNs, was able to restore the human factor IX (hFIX) level to normal physiological values in FIX-k ...[more]
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