Literature-reported formulation composition
Component: CSS-A10 | mol%: 35 | Role: Ionizable lipid
Search the tables and observations reproduced from the product literature guide. Rows may represent measurements, recipes or experimental context; they are not all independent experiments.
Component: CSS-A10 | mol%: 35 | Role: Ionizable lipid
Component: Cholesterol | mol%: 47.5 | Role: Sterol
Component: C14PEG2000 | mol%: 2.5 | Role: PEG lipid
Component: DOTAP | mol%: 15 | Role: Additional lipid components
Component: Reported total | mol%: 100 | Role: Source values retained
Literature-reported Hydrodynamic diameter 80 nm LNP67 characterization Main article Figure 2d
Literature-reported mRNA encapsulation efficiency 86.7% LNP67 characterization Main article Figure 2b
Literature-reported Zeta potential -2.58 mV LNP67 characterization Main article Figure 2e
Literature-reported Apparent LNP pKa 6.4 TNS fluorescence assay Main article Figure 2f and Methods
Literature-reported Functional mRNA delivery to rhesus CD34+ cells Dose-dependent aNLuc expression was detected in CD34+ bone-marrow cells; expression in CD34+ cells was similar to liver-cell expression at both tested doses. Approximately 24 h · Untreated control and liver-cell suspensions Main article Figure 4c-g and accompanying text
Ligand-free bone-marrow delivery LNP67 was selected from an in vivo screen of 105 quality-controlled formulations and showed the most favorable bone-marrow-versus-liver delivery profile among the individually tested lead formulations. Mouse screening and individual confirmation; no antibody or other targeting ligand was attached to LNP67 · Main article Figures 1g and 2a
Mouse HSC/HSPC dose response After intravenous administration, LNP67 produced dose-dependent reporter expression across eight bone-marrow HSC/HSPC subtypes, including reporter-positive multipotent progenitors. C57BL/6J mice; aVHH mRNA; 0.5, 1.0 and 1.5 mg/kg; 16 h · Main article Figure 2i
Preliminary rhesus tolerability observation The authors reported no adverse findings or serum-cytokine differences versus the untreated control at the tested doses without anti-inflammatory pretreatment. Seven treated young rhesus monkeys and one untreated control; 23 serum cytokines; approximately 24 h. This small study is not a formal toxicology package. · Main article Figure 4h and Discussion