An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 26529392.
- Also identified by DOI 10.1021/acs.nanolett.5b03528 and PMC identifier 4869688.
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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.
Medical subject headings
- Lipids
- Nanoparticles
- RNA, Messenger