July 21, 2017
Lipid-like nanoparticles (LLNs) have shown great promise for nucleic acid delivery. Recently, we have developed N1,N3,N5-tris(2-aminoethyl)benzene-1,3,5-tricarboxamide (TT) derived lipid-like compounds, formulated them into TT LLNs for mRNA delivery, and applied an orthogonal array design to facilitate formulation optimization. This chapter focuses on the following contents relevant to lipid-like nanoparticles: formulation method, particle characterization, orthogonal array design, and in vitro assays.
Publication - Summary
Feb 27, 2018
Cell Reports
Hereditary transthryretin amyloidosis is a rare disease caused by mutations in the gene encoding the protein transthyretin (TTR), causing it to misfold into amyloid plaques, leading to debilitating symptoms. In 2018,...
Publication - Abstract
Jun 03, 2017
Molecular Pharmaceutics
Small interfering RNAs (siRNA) have a broad potential as therapeutic agents to reversibly silence any target gene of interest. The clinical application of siRNA requires the use of safe and effective delivery systems. In this study, we investigated...