Table 2
Parameter: Assembly | Reported procedure / limitation: Lipids dissolved in ethanol; RNA in citrate buffer. Pipette mixing for in vitro tests; microfluidic formulation for in vivo experiments and cryo-TEM. | Source: Materials and Methods
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FTT5
Literature records
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.
Table 2
Parameter: Unspecified process values | Reported procedure / limitation: The reviewed protocol does not establish an exact citrate pH, flow ratio, total flow or N/P for this recipe. | Source: Not filled from another lipid protocol
Table 2
Parameter: Optimization outcome | Reported procedure / limitation: Some optimized recipes performed better in vitro but worse in vivo. The original composition was retained for subsequent in vivo work. | Source: SI Figs. S4–S5
Table 2
Parameter: Cargo mapping | Reported procedure / limitation: FVIII mRNA and adenine-base-editor mRNA/sgRNA were studied in separate dose and endpoint experiments. | Source: Main Figs. 3–4
Table 3
Measurement / batch: Original FTT5 LNP | Reported result: Approximately 100 nm; PDI <0.2; zeta potential approximately −11 mV; encapsulation approximately 91% | Evidence context: Main Fig. 2
Table 3
Measurement / batch: Molar-ratio basis | Reported result: The reported entries sum to 90.75 parts | Evidence context: Normalized only for the mass calculation
Table 3
Measurement / batch: Neat-lipid shelf life | Reported result: Not established by formulated-particle or clearance experiments | Evidence context: Follow the supplied batch documentation
Table 4
Experiment and dose: Hepatic reporter expression C57BL/6 mice; intravenous Fluc mRNA 0.5 mg/kg | Readout and comparator: Readout: 6 h Comparator: TT3 formulation | Result and source: Approximately twofold greater liver expression; the reported organ signal is normalized by tissue weight. Main Fig. 2; n=2
Table 4
Experiment and dose: Lipid clearance Intravenous RNA dose 1.8 mg/kg; lipid amounts measured in liver | Readout and comparator: Readout: 20 min, 6 h, 24 h and 48 h Comparator: FTT9 and other study formulations | Result and source: FTT5 retained approximately half of its liver lipid amount at 48 h. The >80% removal at 6 h and complete removal by 24 h belong to FTT9, not FTT5. Main Fig. 2; n=3
Table 4
Experiment and dose: FVIII protein supplementation Wild-type and hemophilia-A mice; FVIII mRNA 2 mg/kg IV | Readout and comparator: Readout: 6 h and 12 h Comparator: Study control groups | Result and source: FVIII concentrations exceeded 140 ng/mL in wild-type mice and 220 ng/mL in hemophilia-A mice at the reported points. These are protein-expression endpoints. Main Fig. 3; n=3
Table 4
Experiment and dose: FVIII functional activity Hemophilia-A mice; same 2 mg/kg FVIII-mRNA study | Readout and comparator: Readout: 6 h and 12 h Comparator: 1 IU/mL defined as 100% activity standard | Result and source: Reported activity approximately 73% and 90%, respectively; not a percentage of corrected liver cells. Main Fig. 3; n=3
Table 4
Experiment and dose: Adenine base editing BALB/c mice; ABE mRNA:sgRNA 1:1 by mass; intravenous total-RNA dose response | Readout and comparator: Readout: Day 7 targeted sequencing Comparator: TT3 comparison and dose-response controls | Result and source: Editing plateaued near 60% at 1 mg/kg. The reported ED50 <0.125 mg/kg describes the dose-response fit, not necessarily 50% absolute editing. Main Fig. 4; n=5; SI Fig. S7
Table 4
Experiment and dose: Short-term histology Hemophilia-A mice; FVIII mRNA 2 mg/kg IV | Readout and comparator: Readout: 72 h Comparator: Study controls | Result and source: No evident abnormalities were reported in examined sections. Repeat-dose, long-term and human tolerability are not established by this endpoint. Main Fig. 3 / SI safety assessment