期刊
NANO LETTERS
卷 15, 期 12, 页码 8099-8107出版社
AMER CHEMICAL SOC
DOI: 10.1021/acs.nanolett.5b03528
关键词
Lipid-like nanoparticles; mRNA delivery; orthogonal array design; hemophilia; genetic disorders
类别
资金
- Early Career Investigator Award from Bayer Hemophilia Awards Program
- Research Reward from Trilink Biotechnologies
- College of Pharmacy at the Ohio State University
- NCI [P30 CA016058]
Systemic delivery of mRNA-based therapeutics remains a challenging issue for preclinical and clinical studies. Here, we describe new lipid-like nanopartides (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 TT3 LLNs, was able to restore the human factor IX (hFIX) level to normal physiological values in FIX-knockout mice. Consequently, development for therapeutic applications. these mRNA based nanomaterials merit further
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