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Literature DB >> 26529392

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.

Bin Li¹, Xiao Luo¹, Binbin Deng², Junfeng Wang¹, David W McComb², Yimin Shi³, Karin M L Gaensler³, Xu Tan⁴, Amy L Dunn^5,6, Bryce A Kerlin^5,6,7, Yizhou Dong¹.

Abstract

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-knockout mice. Consequently, these mRNA based nanomaterials merit further development for therapeutic applications.

Entities: CellLine Chemical Disease Gene Mutation Species

Keywords: Lipid-like nanoparticles; genetic disorders; hemophilia; mRNA delivery; orthogonal array design

Mesh：

Substances：
Lipids
RNA, Messenger

Year: 2015 PMID： 26529392 PMCID： PMC4869688 DOI： 10.1021/acs.nanolett.5b03528

Source DB: PubMed Journal: Nano Lett ISSN： 1530-6984 Impact factor: 11.189

36 in total

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