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 - Abstract
Aug 18, 2020
Nucleic Acids Research
In this paper Alnylam Pharmaceuticals, developer of RNAi therapeutics ONPATTRO® and GIVLAARI®, investigates the biological origins of the extended duration of N-acetylgalactosamine silencing RNA (GalNAc-siRNA) activity observed in vivo.
Publication - Abstract
Jul 19, 2020
Current Topics in Microbiology and Immunology