Table 3
Parameter: Initial screen | Reported procedure / limitation: Pipette mixing; lipid ethanol phase and mRNA in 10 mM citrate, pH 3; ethanol:aqueous 1:3. | Source: Methods: LNP formulation and optimization
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.
Parameter: Initial screen | Reported procedure / limitation: Pipette mixing; lipid ethanol phase and mRNA in 10 mM citrate, pH 3; ethanol:aqueous 1:3. | Source: Methods: LNP formulation and optimization
Parameter: Screening mass recipe | Reported procedure / limitation: Lipidoid:DOPE:cholesterol:DMG-PEG:mRNA = 16:10:10:3:1.6 by mass; reported encapsulation typically 40–80%. | Source: Methods; distinct from molar table
Parameter: Optimized assembly | Reported procedure / limitation: Microfluidic mixing; ethanol:aqueous flow ratio 1:3; 11-10-8:mRNA 10:1 by mass. Total flow and N/P are not reported here. | Source: Methods
Parameter: Purification | Reported procedure / limitation: 20 kDa dialysis against 1× PBS for 2 h; 0.22 µm filtration; stored at 4 °C. | Source: Methods
Parameter: Benchmark recipe | Reported procedure / limitation: MC3 or SM-102 / DSPC / cholesterol / DMG-PEG = 50:10:38.5:1.5; reported MC3:mRNA mass ratio 10:1. | Source: Methods; different helper composition from F5
Measurement / batch: Purified F5 | Reported result: Approximately 71.2 nm; PDI 0.087; encapsulation approximately 95%; zeta potential −1.09 mV | Evidence context: Fig. 4d
Measurement / batch: Apparent LNP pKa | Reported result: 6.22 by TNS assay | Evidence context: Fig. 4 / characterization
Measurement / batch: Recipe discrepancy | Reported result: SI F5 cholesterol 48.5%; Methods text says 48.8, producing a total of 100.3 | Evidence context: SI Table S3 versus Methods; 48.5 is used explicitly for calculations
Experiment and dose: Initial cell screen HepG2; 5,000 cells/well; Fluc mRNA 15 ng/well | Readout and comparator: Readout: 24 h Comparator: Library formulations and assay controls | Result and source: Screening used crude material and the screening recipe. These data are not treated as the purified-F5 outcome. Methods; initial-screen figures
Experiment and dose: Liver reporter expression C57BL/6 mice; intravenous Fluc mRNA 0.1 mg/kg | Readout and comparator: Readout: 4 h Comparator: MC3 LNP | Result and source: F5 gave 4.8-fold higher reported liver luciferase expression. Fig. 5a; n=3, mean ± SD
Experiment and dose: TTR genome editing Female mice; intravenous total RNA 1 mg/kg; Cas9 mRNA:TTR sgRNA 4:1 by mass | Readout and comparator: Readout: Day 7 Comparator: MC3 LNP under the study comparison | Result and source: Approximately 30% versus 7% liver indels; approximately 50% versus 17% serum TTR reduction. Editing and protein reduction are distinct endpoints. Fig. 5c–d; n=5
Experiment and dose: FGF21 expression High-fat-diet obese male mice; intravenous FGF21 mRNA 0.25 mg/kg | Readout and comparator: Readout: Peak at 12 h; exposure followed through 48 h Comparator: MC3 LNP | Result and source: Peak human FGF21 58.8 ± 1.1 ng/mL; approximately fivefold above MC3. Reported 0–48 h exposure AUC was 4.6-fold greater. Fig. 6a; n=4
Experiment and dose: FGF21 treatment High-fat-diet obese mice; intravenous 0.25 mg/kg every other day for three doses | Readout and comparator: Readout: Day 6 and corresponding histology endpoint Comparator: PBS and MC3-based FGF21 arms | Result and source: Reduced body/liver weight and improved liver steatosis were assessed separately; this is a short preclinical treatment study. Fig. 6b–g; n=4
Experiment and dose: siRNA potency Intravenous TTR siRNA dose response, 0.02–1 mg/kg | Readout and comparator: Readout: Day 3 serum TTR Comparator: MC3 LNP | Result and source: Reported ED50 approximately 0.029 versus 0.030 mg/kg: comparable siRNA potency, despite the mRNA advantage. SI siRNA experiment; main Discussion